Questions List
Why diserythriopoisis can not cope with demands of person for oxygen supply dr khalid pakistan Posted onMarch 9, 2021 12:06 pm
Valeria, Nica and Stuart: Thank you very much for this great e-conference!!
Great work!! (M. Ostuni, from Paris)
Posted onMarch 7, 2021 5:48 pm
Excellent e-conference, great speakers and presentations. Looking forward to the next one, hopefully in person.
Laura Breda, CHOP, PhiladelphiaPosted onMarch 7, 2021 5:46 pm
Thank you very much for an excellent conferencePosted onMarch 7, 2021 5:43 pm
Glutamine concentration in blood plasma of healthy people is about 0.6 mM. What's about MDS affected patientsPosted onMarch 7, 2021 5:31 pm
A question for Lucia De Franceschi. Could we expect that glutamine will also increase erythroid lineage commitment (as reported by N. Taylor's team)? Could we think to combine glutamine and HU?
M. Ostuni (Paris)
Posted onMarch 7, 2021 5:17 pm
Do you think that the simultaneous use of iron chelation and luspatercept is useful to improve Hb levels in patients with Ringed and non erythroblastic refratory anaemia?Posted onMarch 7, 2021 5:14 pm
Question to Dr Jarocha. Could you please elaborate a bit more on the mechanism by which this vector suppresses the production of endogenous "mutant" b-globin gene? Georgios Kaltsounis, GreecePosted onMarch 7, 2021 5:12 pm
For Danuta Jarocha: how do you obtain separation of b-globin and anti-BCL11A rna? ThanksPosted onMarch 7, 2021 5:12 pm
Question for Tomas Ganz:
You mentioned HIF prolyl -hydroxylase as imminent therapy for CKD. Do you see a role of this oral therapy in other disorders with ineffective erythropoiesis such as SCD or thalassemia and can HIF prolyl -hydroxylase stimulate HbF?
Posted onMarch 7, 2021 4:59 pm
How to choose between these different new treatments in SCD since we have no phase III trials using these different drugs together?
Bernard Drénou Mulhouse France
Posted onMarch 7, 2021 4:58 pm
For Dr. Platzbecker. Very nice talk. Could you share more information about the role of Luspatercept in mesenchymal cells? Thank you, Stefano Rivella-CHOP-USAPosted onMarch 7, 2021 4:48 pm
What is the mechanism of EPO increase in patients treated with luspatercetp ?
Clara Camaschella MilanoPosted onMarch 7, 2021 4:44 pm
Prof. Ganz, thanks for your presentation, very clear. Do you see any potential for a more intense iron chelation therapy to improve ineffective erythropoiesis of iron loading anemias?Posted onMarch 7, 2021 4:41 pm
Thank you for your talk.
What about the concerns of stabilising HIF by PHI due to pleiotropic effects of HIF itself?
Clara Camaschella MilanoPosted onMarch 7, 2021 4:34 pm
Laura Silvestri, Milano
Outstanding presentation! My question is related to the effect of iron deficiency on scribble. Scribble is ubiquitously expressed and is a master regulator of receptor trafficking and signaling. Do you think iron deficiency downregulates scribble non only in erythroid precursors, as shown by in vitro experiments, but also in other cells and tissues? Posted onMarch 7, 2021 4:29 pm
**** Session 9 *******Posted onMarch 7, 2021 4:15 pm
Posted onMarch 7, 2021 4:02 pm
For dr bejar: having heard about increase of MDS in sickle cell anemia, what is the rate of clonal hematopoiesis and somatic mutations in this disease?
Posted onMarch 7, 2021 4:00 pm
From Emery Bresnick to Mitch and others, This might have been addressed, but can you touch on the state of the art for non-viral delivery methods for genetic modifications in the context of hemoglobinopathies, e.g. strictly RNA-based delivery mechanisms and/or emerging novel approaches.
Posted onMarch 7, 2021 3:57 pm
What is the basis of higher risk of developing MDS and AML in sickle cell anemia vs thalassemia?Posted onMarch 7, 2021 3:53 pm
Karina Tozatto Maio, Brazil, to all speakers
How would you assess pre gene therapy (editing/LV addition) safety in patients with SCD, considering that they might be at higher risk of MDS/AML development? Would you consider performing MDS/AML sequencing panels in young adults with SCD before manipulating the cells?Posted onMarch 7, 2021 3:52 pm
yes i am happy with the answer, thank you (Han Verhagen) Posted onMarch 7, 2021 3:50 pm
Q to Marina: great update. Are you happy with Busulfan as conditioning agent? Why not use the low dose TBI with/without Campath?Posted onMarch 7, 2021 3:44 pm
For Dr Bejar: do you think that CAR-T therapy could be ever programmed for all MDS types, for instance, lower risk MDS ? or it will be only dedicated to MDS with marrow blasts ? Given the difficulties in characterizing the MDS initiating clone it seems one of the most difficult disease to targetPosted onMarch 7, 2021 3:43 pm
Han Verhagen (Sanquin, Amsterdam , Netherlands)
Question to Mitchel Weiss
i was surprised on the data you showed with the FISH results, 2.5% of genomic abnormailites is a lot. Can you tell us a bit more on what kind of technique you used in these experiments and what concentrations? was it RNP nucleofection, or AAV6 mediated editing? Did you try to use a high Fidelity Cas9, are the results different? Do you see it for all sgRNA's or did you only test one? Posted onMarch 7, 2021 3:36 pm
Posted onMarch 7, 2021 3:29 pm
Posted onMarch 7, 2021 3:29 pm
Are there developments on in vivo delivery of GT?Posted onMarch 7, 2021 3:21 pm
Thank you for your excellent presentations. Question to Dr Cavazzana: what is today the clinical course of the first patient that underwent gene therapy in the first clinical trial in Paris in 2006? That clonal expansion and HMGA2 activation has subsided? Is he still transfusion free? Do you see any connection between that clonal expansion and the SAE observed in the SCD patients lately? Georgios Kaltsounis, GreecePosted onMarch 7, 2021 3:11 pm
Posted onMarch 7, 2021 3:06 pm
Sorry, not sure my question was submitted/internet issues. In light of the recent data with bluebird bio (MDS and AML), is it possible that SCD patients may have preleukemic lesions or clonal hematopoiesis? The chronic inflammation in CD patients may increased proliferation of HSC, accelerates somatic evolution and clonal hematopoiesis. Is this is true, how this will change the clinical trial using autologous HSC? Could this be relevant also for gene editing trials? Thank you, Stefano Rivella, CHOP, USAPosted onMarch 7, 2021 3:05 pm
Posted onMarch 7, 2021 3:04 pm
In light of recent data from bluebird bio in SCD patients, can you speculate if the autologous cells may have preleukemic lesions or clonal hematopoiesis, that the process of mobilization and culture condition may exacerbate? The HSC of SCD patients may be subjected to chronic inflammation leading to increased proliferation, which would accelerate somatic evolution and clonal hematopoiesis. If this is the case, how these trials should be modified? Could the same problem be relevant for gene editing trials?Posted onMarch 7, 2021 3:01 pm
Can you speculate on the potential proportion of cells non corrected and how this may still contribute to ineffective erythropoiesis despite hemoglobin levels in the range of 10 after gene therapy? Is this irrelevant or may have long term consequences? Stefano Rivella-Philadelphia-USAPosted onMarch 7, 2021 2:53 pm
**** Session 8 ****Posted onMarch 7, 2021 2:53 pm
Posted onMarch 7, 2021 2:14 pm
Do you have access in Giordan to new therapeutic approaches such as gene therapy or Luspatercept therapyPosted onMarch 7, 2021 2:04 pm
where does Luspatercept in the treatment of CDA?Posted onMarch 7, 2021 2:04 pm
Anne Grete From Norway:
Wold you all recommend HU and hypertransfusion prior to SCT in TM and SCA?Posted onMarch 7, 2021 2:02 pm
for dr Shimamura: is there any early sign or symptom which can justify molecular analysis for predisposing risk?Posted onMarch 7, 2021 2:02 pm
Emery Bresnick for Lydia:
Comment: Nice presentation. I believe you referred to GATA1 as not related to ribosome biogenesis. GATA1 regulates the Exosome Complex that is crucial for rRNA processing/generation and is therefore intimately linked to ribosome structure/function. It would not be surprising if this activity in conjunction with additional activities contribute to the compromised erythroblasts.Posted onMarch 7, 2021 2:01 pm
Emery Bresnick for Akiko:
Very nice presentation:
1. Were there SDS cases in which there was a conspicuous lack of somatic mutations that could be implicated in disease progression and if so did you probe for potential non-coding genetic variation?
2. You described the deleterious impact of p53 somatic mutations. Did you detect other somatic mutations with any interesting frequency that have been implicated in triggering and or progression of pathogenesis for other MDS/AML-predisposing germline mutations.Posted onMarch 7, 2021 2:00 pm
With these new understanding of DBA, is there any new therapeutic approach?Posted onMarch 7, 2021 1:36 pm
Dr Russo: sometime the work up of hemolytic anemias (excluding the immune types) is difficult and especially is requiring long time before getting a response. Do you suggest to perform a specific panel NGS directly at first step? is it cost-effective?Posted onMarch 7, 2021 1:26 pm
Thanks for nice presentation. How to Differentiate unilinage MDS from CDAII?
Dr. Datta BotswanaPosted onMarch 7, 2021 1:22 pm
Posted onMarch 7, 2021 1:21 pm
Posted onMarch 7, 2021 1:21 pm
Roberta great talk!
Do you think that small doses of hepcidin analog could be beneficial in some types of CDAs, especially in those with ERFE variant?
Joe PrchalPosted onMarch 7, 2021 1:21 pm
****** Day 3************Posted onMarch 7, 2021 1:04 pm
I need an update on gene therapy in sickle cell disease Posted onMarch 7, 2021 11:40 am
Posted onMarch 6, 2021 6:43 pm
Posted onMarch 6, 2021 6:43 pm
Posted onMarch 6, 2021 6:27 pm
Velia Fowler: Do you think E-cadherin in erythroblasts interacts with E-cadherin in other erythroblasts, or in other cell types?
Posted onMarch 6, 2021 6:23 pm
Great session!
FrancescaPosted onMarch 6, 2021 6:00 pm
Last question is a good title for a long talkPosted onMarch 6, 2021 5:58 pm
but inhibition of protein synthesis by phosphorylated eIF2a occurs in early erythroblasts before the massive globin synthesis. So I think that contributes to overall inhibition of erythropoiesis Posted onMarch 6, 2021 5:57 pm
Clara puoi ricordare the poster walk e le 2 meet the expert sessions
graziePosted onMarch 6, 2021 5:57 pm
To Martina
Great talk, thanks.
It is known that Iron could also be a growth factor for tumor cells
I am wondering whether in your model, tumor cells can take up iron nanoparticles or if they accumulate specifically in macrophages.Posted onMarch 6, 2021 5:56 pm
Thank you Clara and thank you all the speakers. Excellent session
NicaPosted onMarch 6, 2021 5:55 pm
It was Ella :)
Posted onMarch 6, 2021 5:53 pm
Posted onMarch 6, 2021 5:51 pm
and is reduced protein synthesis then the cause of small ery's
Posted onMarch 6, 2021 5:51 pm
Posted onMarch 6, 2021 5:51 pm
achillePosted onMarch 6, 2021 5:51 pm
What is the role for low overall protein production by HRI-mediated phosphorylation of eIF2a
'marieke von Lindern Posted onMarch 6, 2021 5:51 pm
why DMT1 cells deficient a re so little
Posted onMarch 6, 2021 5:51 pm
Great job iron ladies, this was a wonderful session!Posted onMarch 6, 2021 5:48 pm
not a question, but my congratulations to all the speakers of this session. Patricia Aguilar Martinez
(France)Posted onMarch 6, 2021 5:47 pm
Posted onMarch 6, 2021 5:46 pm
Posted onMarch 6, 2021 5:46 pm
Posted onMarch 6, 2021 5:46 pm
clara le domande erano le mie: achille
Posted onMarch 6, 2021 5:46 pm
To Porto: Do you have limitation on using EV iron in some microcytic anemia or in inflammatory anemias?
Posted onMarch 6, 2021 5:45 pm
How is the KER drug different from other activin trap drugs?Posted onMarch 6, 2021 5:42 pm
Posted onMarch 6, 2021 5:41 pm
Posted onMarch 6, 2021 5:41 pm
Posted onMarch 6, 2021 5:41 pm
For Dr. Porto. Very nice data and presentation. Not sure if I understood the phenotype of the thalassemic patient. I think you mentioned he was transfusion dependent but then he benefit from phlebotomy. How did he manage the anemia? Thanks Stefano Rivella-Philly-USPosted onMarch 6, 2021 5:39 pm
To Graca : atypical for you means that is not monogenic ? or is related to what we know in the more common casePosted onMarch 6, 2021 5:37 pm
For Dr. Muckenthaler. Great talk. Can you polarized M2 to M1macrophages using specific cytokines instead of iron? Stefano Rivella-Philly-USAPosted onMarch 6, 2021 5:36 pm
For Laura Silvestri: could you comment on the role of monoferric vs diferric transferric in regulating hepcidin, and how the ratio of monoferric vs diferric transferrin may change with stimulation of erythropoieisis?Posted onMarch 6, 2021 5:36 pm
For Dr. Feigenson: sotatercept (another activin II ligand trap) did not show very promising results. do you expect better results for your molecule? in which aspects is it different from luspatercept and sotatercept? Also, are you planning studies on thalassemias? thanks a lotPosted onMarch 6, 2021 5:35 pm
To Dr. Feigenson: How does the KER drug regulate erythropoietin production?Posted onMarch 6, 2021 5:35 pm
For Dr. Feigenson. Exciting data How Ker-050 is different from Luspatercept? Any insight in the target molecules? Thanks you. Stefano Rivella-Philadelphia-USA To all: wonderful sessionPosted onMarch 6, 2021 5:34 pm
Posted onMarch 6, 2021 5:34 pm
To Marina:
Did you explore a thalassemia model?
Antonio PigaPosted onMarch 6, 2021 5:34 pm
Posted onMarch 6, 2021 5:34 pm
Posted onMarch 6, 2021 5:34 pm
Posted onMarch 6, 2021 5:33 pm
For Prof. SIlvestri: great presentation, very inspiring! my question is: you showed that circulating iron decreases EPO production in the kidney via IRP1. what do you think is the biological significance of this mechanism? and how is it linked to ineffective erythropoiesis, where on the contrary persistent anemia causes increased levels of EPO, even in presence of iron overload? ThanksPosted onMarch 6, 2021 5:22 pm
To Dr Muckenthaler. Thank you for this talk. Tumor immune microenvironment allows to classify solid tumors and their prognosis depending on the presence of inflammatory M2 macrophages or a more predominant non-inflammatory invasion by lymphocytes. Did you have the opportunity to investigate whether injection of iron nanoparticles to non inflammatory tumors induced tumor cell apoptosis?Posted onMarch 6, 2021 5:18 pm
iron iOS important in all the cells of the body, however the quantity is very different from erythroid and nervous cells (for example) the mechanism of controls is always the same?
Posted onMarch 6, 2021 4:41 pm
Maria you use epoetina or darbo in elderly patients? And High dose aswell? What do you think about the tromboembolic risco associated? Thank you.Posted onMarch 6, 2021 4:05 pm
************ Sympo *********Posted onMarch 6, 2021 3:39 pm
Can you explain the relation between NfK inhibition eryptosis and obesityPosted onMarch 6, 2021 3:07 pm
How does your gluco mechanism compare with the mechanisms described by Lodish and colleagues involving PPARa and other factors/pathwaysPosted onMarch 6, 2021 3:04 pm
Is mitapivat (piravute kinase activator) acting decreasing eryptosis, too?
Posted onMarch 6, 2021 3:04 pm
Can you retrieve the cultured cells from the sponge model ?
Posted onMarch 6, 2021 3:00 pm
Balduini: Are you using completely synthetic media? e.g. PVA to replace serum albumin?Posted onMarch 6, 2021 2:59 pm
Have you compared the proteomes from early RBCs ans RBCs going erypthosis ? F. Baklouti, Lyon, FrancePosted onMarch 6, 2021 2:57 pm
setting a 3d BM model from a patient affected by a hematological hereditary disorder could you manipulateor treat the cells to correct the disorderPosted onMarch 6, 2021 2:55 pm
Are the platelets produced in the BM model viable and functional; could your model be use to produce erythrocytes and platelets?Posted onMarch 6, 2021 2:48 pm
Thank you, Alessandra, for this talk. This a very interesting and exciting approach. I have a technical question: how do you control molecular gradients in time and space? Is flow system enough efficient?
Mariano (Paris)
Posted onMarch 6, 2021 2:45 pm
in DBA, there is a cell cycle arrest in Go/G1 with less cells in phase S, do you think it may interferfer in the response of CFU-e proliferation between cord blood versus peripheral blood in DBA patient. difficult to collect cord blood from DBA patient and then follow the culture from the peripheral blood from the same patient. but it will be interesting to look if there is also some differences in that case.
Posted onMarch 6, 2021 2:35 pm
Thank you for your talk. What are the mechanisms involved in cytokines uptake inside RBC? Are this mechanisms specific of RBC? What is the final fate of cytokines inside RBC? Mariano (Paris)Posted onMarch 6, 2021 2:29 pm
very nice talk lionel. congrats. my question: when we were doing only culture in methylcellulose with or without DXM in CD34+ from peripheral blood from DBA patients, some patients with good DXM response in vivo responded as well in vitro to DXM with increased proliferation but some don't suggesting no correlation between in vivo and vitro response to DXM in patient (reverse was true as well, bad response in vitro and good response in vivo). have you noticed as well some discrepancies in your experience like that with p57 evaluation? and perhaps it will be interesting to find some patients with this kind of discrepancy and look at p57 and so to identify why this lack of correlation between in vivo in vitro in some cases (new pathways? exogenous or endogenous modifiers?)? all the best and thanks for the teasing for tomorrowPosted onMarch 6, 2021 2:28 pm
Lionel, great talk. What is the mechanism of p57 induction by dex in adult erythroid progenitors.
Michaela FontenayPosted onMarch 6, 2021 2:18 pm
Thanks for this talk.
Do you know whether covid-19 patients treated with dex have any complication related to progenitors’ proliferation? Specially these in intensive care.
Posted onMarch 6, 2021 2:17 pm
Jonas Schulte-Schrepping, Bonn: Thanks for taking up my rather long questions and answering them in so much detail. Super interesting and fascinating insights!Posted onMarch 6, 2021 1:51 pm
Sorry, I forgot to add my name and country to the questions to Prof Sankaran before: Jonas Schulte-Schrepping, Bonn Germany, Speaker in Session 1 yesterday. I have another Question to Prof. Sankaran with regard to the multiome solution from 10X. Did you observe the cells of one clone of terminally differentiated hematopoietic cells to be rather homogeneous in their transcriptome or spanning a broad range of phenotypes. And how were these observations reflected in the open chromatin profiles?Posted onMarch 6, 2021 1:44 pm
Posted onMarch 6, 2021 1:37 pm
Hi, I am Siyu from Oxford, here is my question for Dr. Sankaran: in the single cell ATAC-seq, you fixed the cells with 1% formaldehyde prior lysis and tagmentation to reduce contamination, I was wondering is there any difference in terms of results compared to standard scATAC-seq without the fixation step?Posted onMarch 6, 2021 1:32 pm
To Prof. Sankaran: This question might be rather unintuitive giving the high value of mitochondrial reads in your analyses. But, since mitochondrial reads in scRNA-seq often take up a lot of the generated reads and do not always contain relevant information given the given biological question and your extensive work on those reads, did you also find a way how to reduce those reads from scRNA-seq libraries to leave more space for more informative transcripts?Posted onMarch 6, 2021 1:23 pm
Did you already test your approach of lineage tracing/clonality analysis using the new combined scRNA+ATAC-seq methodology from 10X? This should allow to match phenotype and clonality even better. Posted onMarch 6, 2021 1:19 pm
Posted onMarch 6, 2021 1:13 pm
Posted onMarch 6, 2021 1:13 pm
Laura Silvestri, Milan
What is the mechanism of erythropoiesis improvement by vitamin B6 administration in R411H transgenic mice?Posted onMarch 6, 2021 12:48 pm
besides ineffective erythropoiesis is there a reduction of life span of siderocytes?
Clara Camaschella - MilanoPosted onMarch 6, 2021 12:48 pm
********** Day 2 ********Posted onMarch 6, 2021 12:37 pm
Posted onMarch 6, 2021 12:34 pm
Are there any contraindications to the SARS-CoV-2 vaccine for patients with G6PD deficiency due to vaccine components?Posted onMarch 6, 2021 11:52 am
Are there any contraindications to the SARS-CoV-2 vaccine for patients with G6PD deficiency due to vaccine components?Posted onMarch 6, 2021 11:50 am
Posted onMarch 6, 2021 8:53 am
Thank you Ismael
Posted onMarch 5, 2021 6:39 pm
Thanks for the talk by Ismael, can I ask is there any possibility to change the codon composition to increase the translation rate for RPS14 deficient patients? Have you tried this? Thanks. Posted onMarch 5, 2021 6:28 pm
Is it possible to decrease ROS to improve ineffective erythropoiesis?Posted onMarch 5, 2021 6:27 pm
Marc, Are there unique markers for the mesenchymal cells that talk to erythroid cells vs. more general mesenchymal cells in the bone marrow?
Emery Bresnick, WIPosted onMarch 5, 2021 6:26 pm
Ismael, Nice presentation. Does the GATA1 translational downregulation impact a small fraction of GATA1-regulated genes/proteins or a highly restricted cohort?
emery bresnick WIPosted onMarch 5, 2021 6:24 pm
Why MDS LOW RISK AS REFRACTORY ANEMIA OR RARS WHICH CHARACTERISED BY INEFFECTIVE ERYTHROPOIESIS DO NOT PRESENT SO COMMON EXTRAMEDULARY ERYTHROPOIESIS AS THALASSEMIA INDERMEDIA? Posted onMarch 5, 2021 5:58 pm
Posted onMarch 5, 2021 5:56 pm
What about the controversy of the data of GDF11 erythroid KO mice that respond as wild type to action ligand trap?
Clara Camaschella, MilanoPosted onMarch 5, 2021 5:56 pm
How do you explain the effect of Luspatercept in beta0/beta0 thalassemia patients?Posted onMarch 5, 2021 5:53 pm
Laura Silvestri, Milano
In vivo studies exclude GDF11 as a player in ineffective erythropoiesis and the ligand sequestered by ACE molecules.
What about GDF8? There is any role for this ligand in ineffective erythropoiesis?Posted onMarch 5, 2021 5:47 pm
******** Session 3 **********Posted onMarch 5, 2021 5:34 pm
Do SF3B1 mutations affect splicing and translation of key erythroid transcription factors?Posted onMarch 5, 2021 5:34 pm
Hi all, what do you think about the recently published paper 'Human erythroid progenitors are directly infected by SARS-CoV-2: implications for emerging erythropoiesis in severe COVID19 patients' on Stem Cell Reports about a specific population of herythrocytes being more susceptible for infection? Thanks.Posted onMarch 5, 2021 5:03 pm
hola buen dia soy el dr meza de hetaologia en mexico, seguimos encontrando linfopenia severa con neutrofilia como una de las cosas mas frecuentes en paciente en estado critico en mexico con esteroides a dosis bajas dexametasona 6-8 mg cada 24 hrs , ¿creen que tenga un beneficio aumentar la dosi de esteroide a 24 mgs ) o pulos de metilpredinoslona 500 mgs cada 24 hrs ?Posted onMarch 5, 2021 4:55 pm
For Prof. Tolosano and Motta: could you comment about any possible connection among your data, specifically is there a role of heme methabolism in explaining covid response observed among beta-thalassemia patients? And what about SCD?
ThanksPosted onMarch 5, 2021 4:44 pm
May I miss this. Italy Blood bank may have done routinarlly any PCR Covid Test for assymptomatic blood donors ?Posted onMarch 5, 2021 4:43 pm
Posted onMarch 5, 2021 4:43 pm
Please can we get this lecture as PowerPoint Posted onMarch 5, 2021 4:41 pm
Marieke von Lindern : Covid patients complain about prolonged fatigue (also after relatively mild symptoms) is this linked to Hb and hemoglobin?Posted onMarch 5, 2021 4:03 pm
Posted onMarch 5, 2021 4:02 pm
to Hal Drakesmith by Marieke von Lindern (Sanquin, Amsterdam):
Does the axes of IL6-hepcidin-low iron regulate the proliferation of the lymphoid cells of the adaptive immune in general (suggested by the model on your conclusion slide), or does iron availability much more affect the balance in activity between T effector cells and T regulatory cells ? Posted onMarch 5, 2021 3:53 pm
Laura Silvestri, Milan
Fe/S cluster and heme biosynthesis are two processes closely interconnected. Fe/s clusters are prosthetic groups for enzymes of the respiratory chain and krebs cycle. Is the Fe/S cluster machinery affected by FLVCR1a silencing or overexpression. Do you think this biosynthetic pathway can partially explain the cell phenotype you described?Posted onMarch 5, 2021 3:44 pm
A pediatric disease named IRIDA causes hypoferremia due to constitutively high hepcidin levels. I was wondering whether an impaired immune response after vaccination has been described for these patients.Posted onMarch 5, 2021 3:34 pm
Clara Camaschella, Milano
As for anti COVId vaccine is there any evidence of different response of iron deficient individuals?Posted onMarch 5, 2021 3:32 pm
Are the presentations recorded so that I can look back?Posted onMarch 5, 2021 3:05 pm
************* Session 2 ********Posted onMarch 5, 2021 3:01 pm
Posted onMarch 5, 2021 1:58 pm
What's the difference between these immature Neutrophils and MDSCs?Posted onMarch 5, 2021 1:52 pm
What is the difference between the immature neutrophils and MDSCs?
Posted onMarch 5, 2021 1:47 pm
Petros Papadopoulos/Madrid
Was there any platelet gene expression signature in the COVID patients in whole blood RNAseq?Posted onMarch 5, 2021 1:47 pm
Verdier Frédérique (france) for Dr Nai:
A question about the recovery of anemia in beta thalassemia in tfr2BM KO mice. Is it possible that the absence of TFR2 in erythroblasts inhibit erythroid maturation, that consequently induce stress erythropoiesis and mobilization of iron in excess in this pathology ? Posted onMarch 5, 2021 1:42 pm
globin editing speaker:
Can you discuss immunogenicity considerations with the in vivo editing strategy and if relevant, are unique measures taken to mitigate immune-related issues?
emery bresnick, WIPosted onMarch 5, 2021 1:39 pm
TO Dr. Schrepping: is there any therapeutic approach that may counteract COVID-related emergency hemopoiesis?
Posted onMarch 5, 2021 1:35 pm
Posted onMarch 5, 2021 1:32 pm
for CasanovaPosted onMarch 5, 2021 1:32 pm
Considering the battery of functionally significant genetic variants demonstrated to cause or contribute to primary immunodeficiency disease, did the Covid patient sequencing reveal the majority of all of these variants or were certain variants conspicuously absent
emery bresnick, WIPosted onMarch 5, 2021 1:32 pm
For Ms Georgakopoulou:
Thank you for the presentation. Have you checked if there is any translocation in your edited HSCs, as you target more than 1 target?
Have you also thought to replace the Cas9 with base editors in order to avoid any such event, if present?
Thank you,
Panagiotis Antoniou, IMAGINE Institute, Paris, FrancePosted onMarch 5, 2021 1:28 pm
Posted onMarch 5, 2021 12:49 pm
Laura Silvestri, Milan
What is the frequency of inborn errors in Interferon-gamma genes in the general population?
Posted onMarch 5, 2021 12:48 pm
123Posted onMarch 5, 2021 8:12 am
TestsssPosted onMarch 5, 2021 8:11 am
********Day 1********Posted onMarch 5, 2021 7:58 am
test 123Posted onMarch 3, 2021 7:28 pm
Why diserythriopoisis can not cope with demands of person for oxygen supply dr khalid pakistan Posted onMarch 9, 2021 12:06 pm
Valeria, Nica and Stuart: Thank you very much for this great e-conference!! Great work!! (M. Ostuni, from Paris) Posted onMarch 7, 2021 5:48 pm
Excellent e-conference, great speakers and presentations. Looking forward to the next one, hopefully in person. Laura Breda, CHOP, PhiladelphiaPosted onMarch 7, 2021 5:46 pm
Thank you very much for an excellent conferencePosted onMarch 7, 2021 5:43 pm
Glutamine concentration in blood plasma of healthy people is about 0.6 mM. What's about MDS affected patientsPosted onMarch 7, 2021 5:31 pm
A question for Lucia De Franceschi. Could we expect that glutamine will also increase erythroid lineage commitment (as reported by N. Taylor's team)? Could we think to combine glutamine and HU? M. Ostuni (Paris) Posted onMarch 7, 2021 5:17 pm
Do you think that the simultaneous use of iron chelation and luspatercept is useful to improve Hb levels in patients with Ringed and non erythroblastic refratory anaemia?Posted onMarch 7, 2021 5:14 pm
Question to Dr Jarocha. Could you please elaborate a bit more on the mechanism by which this vector suppresses the production of endogenous "mutant" b-globin gene? Georgios Kaltsounis, GreecePosted onMarch 7, 2021 5:12 pm
For Danuta Jarocha: how do you obtain separation of b-globin and anti-BCL11A rna? ThanksPosted onMarch 7, 2021 5:12 pm
Question for Tomas Ganz: You mentioned HIF prolyl -hydroxylase as imminent therapy for CKD. Do you see a role of this oral therapy in other disorders with ineffective erythropoiesis such as SCD or thalassemia and can HIF prolyl -hydroxylase stimulate HbF? Posted onMarch 7, 2021 4:59 pm
How to choose between these different new treatments in SCD since we have no phase III trials using these different drugs together? Bernard Drénou Mulhouse France Posted onMarch 7, 2021 4:58 pm
For Dr. Platzbecker. Very nice talk. Could you share more information about the role of Luspatercept in mesenchymal cells? Thank you, Stefano Rivella-CHOP-USAPosted onMarch 7, 2021 4:48 pm
What is the mechanism of EPO increase in patients treated with luspatercetp ? Clara Camaschella MilanoPosted onMarch 7, 2021 4:44 pm
Prof. Ganz, thanks for your presentation, very clear. Do you see any potential for a more intense iron chelation therapy to improve ineffective erythropoiesis of iron loading anemias?Posted onMarch 7, 2021 4:41 pm
Thank you for your talk. What about the concerns of stabilising HIF by PHI due to pleiotropic effects of HIF itself? Clara Camaschella MilanoPosted onMarch 7, 2021 4:34 pm
Laura Silvestri, Milano Outstanding presentation! My question is related to the effect of iron deficiency on scribble. Scribble is ubiquitously expressed and is a master regulator of receptor trafficking and signaling. Do you think iron deficiency downregulates scribble non only in erythroid precursors, as shown by in vitro experiments, but also in other cells and tissues? Posted onMarch 7, 2021 4:29 pm
**** Session 9 *******Posted onMarch 7, 2021 4:15 pm
Posted onMarch 7, 2021 4:02 pm
For dr bejar: having heard about increase of MDS in sickle cell anemia, what is the rate of clonal hematopoiesis and somatic mutations in this disease? Posted onMarch 7, 2021 4:00 pm
From Emery Bresnick to Mitch and others, This might have been addressed, but can you touch on the state of the art for non-viral delivery methods for genetic modifications in the context of hemoglobinopathies, e.g. strictly RNA-based delivery mechanisms and/or emerging novel approaches. Posted onMarch 7, 2021 3:57 pm
What is the basis of higher risk of developing MDS and AML in sickle cell anemia vs thalassemia?Posted onMarch 7, 2021 3:53 pm
Karina Tozatto Maio, Brazil, to all speakers How would you assess pre gene therapy (editing/LV addition) safety in patients with SCD, considering that they might be at higher risk of MDS/AML development? Would you consider performing MDS/AML sequencing panels in young adults with SCD before manipulating the cells?Posted onMarch 7, 2021 3:52 pm
yes i am happy with the answer, thank you (Han Verhagen) Posted onMarch 7, 2021 3:50 pm
Q to Marina: great update. Are you happy with Busulfan as conditioning agent? Why not use the low dose TBI with/without Campath?Posted onMarch 7, 2021 3:44 pm
For Dr Bejar: do you think that CAR-T therapy could be ever programmed for all MDS types, for instance, lower risk MDS ? or it will be only dedicated to MDS with marrow blasts ? Given the difficulties in characterizing the MDS initiating clone it seems one of the most difficult disease to targetPosted onMarch 7, 2021 3:43 pm
Han Verhagen (Sanquin, Amsterdam , Netherlands) Question to Mitchel Weiss i was surprised on the data you showed with the FISH results, 2.5% of genomic abnormailites is a lot. Can you tell us a bit more on what kind of technique you used in these experiments and what concentrations? was it RNP nucleofection, or AAV6 mediated editing? Did you try to use a high Fidelity Cas9, are the results different? Do you see it for all sgRNA's or did you only test one? Posted onMarch 7, 2021 3:36 pm
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Are there developments on in vivo delivery of GT?Posted onMarch 7, 2021 3:21 pm
Thank you for your excellent presentations. Question to Dr Cavazzana: what is today the clinical course of the first patient that underwent gene therapy in the first clinical trial in Paris in 2006? That clonal expansion and HMGA2 activation has subsided? Is he still transfusion free? Do you see any connection between that clonal expansion and the SAE observed in the SCD patients lately? Georgios Kaltsounis, GreecePosted onMarch 7, 2021 3:11 pm
Posted onMarch 7, 2021 3:06 pm
Sorry, not sure my question was submitted/internet issues. In light of the recent data with bluebird bio (MDS and AML), is it possible that SCD patients may have preleukemic lesions or clonal hematopoiesis? The chronic inflammation in CD patients may increased proliferation of HSC, accelerates somatic evolution and clonal hematopoiesis. Is this is true, how this will change the clinical trial using autologous HSC? Could this be relevant also for gene editing trials? Thank you, Stefano Rivella, CHOP, USAPosted onMarch 7, 2021 3:05 pm
Posted onMarch 7, 2021 3:04 pm
In light of recent data from bluebird bio in SCD patients, can you speculate if the autologous cells may have preleukemic lesions or clonal hematopoiesis, that the process of mobilization and culture condition may exacerbate? The HSC of SCD patients may be subjected to chronic inflammation leading to increased proliferation, which would accelerate somatic evolution and clonal hematopoiesis. If this is the case, how these trials should be modified? Could the same problem be relevant for gene editing trials?Posted onMarch 7, 2021 3:01 pm
Can you speculate on the potential proportion of cells non corrected and how this may still contribute to ineffective erythropoiesis despite hemoglobin levels in the range of 10 after gene therapy? Is this irrelevant or may have long term consequences? Stefano Rivella-Philadelphia-USAPosted onMarch 7, 2021 2:53 pm
**** Session 8 ****Posted onMarch 7, 2021 2:53 pm
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Do you have access in Giordan to new therapeutic approaches such as gene therapy or Luspatercept therapyPosted onMarch 7, 2021 2:04 pm
where does Luspatercept in the treatment of CDA?Posted onMarch 7, 2021 2:04 pm
Anne Grete From Norway: Wold you all recommend HU and hypertransfusion prior to SCT in TM and SCA?Posted onMarch 7, 2021 2:02 pm
for dr Shimamura: is there any early sign or symptom which can justify molecular analysis for predisposing risk?Posted onMarch 7, 2021 2:02 pm
Emery Bresnick for Lydia: Comment: Nice presentation. I believe you referred to GATA1 as not related to ribosome biogenesis. GATA1 regulates the Exosome Complex that is crucial for rRNA processing/generation and is therefore intimately linked to ribosome structure/function. It would not be surprising if this activity in conjunction with additional activities contribute to the compromised erythroblasts.Posted onMarch 7, 2021 2:01 pm
Emery Bresnick for Akiko: Very nice presentation: 1. Were there SDS cases in which there was a conspicuous lack of somatic mutations that could be implicated in disease progression and if so did you probe for potential non-coding genetic variation? 2. You described the deleterious impact of p53 somatic mutations. Did you detect other somatic mutations with any interesting frequency that have been implicated in triggering and or progression of pathogenesis for other MDS/AML-predisposing germline mutations.Posted onMarch 7, 2021 2:00 pm
With these new understanding of DBA, is there any new therapeutic approach?Posted onMarch 7, 2021 1:36 pm
Dr Russo: sometime the work up of hemolytic anemias (excluding the immune types) is difficult and especially is requiring long time before getting a response. Do you suggest to perform a specific panel NGS directly at first step? is it cost-effective?Posted onMarch 7, 2021 1:26 pm
Thanks for nice presentation. How to Differentiate unilinage MDS from CDAII? Dr. Datta BotswanaPosted onMarch 7, 2021 1:22 pm
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Roberta great talk! Do you think that small doses of hepcidin analog could be beneficial in some types of CDAs, especially in those with ERFE variant? Joe PrchalPosted onMarch 7, 2021 1:21 pm
****** Day 3************Posted onMarch 7, 2021 1:04 pm
I need an update on gene therapy in sickle cell disease Posted onMarch 7, 2021 11:40 am
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Velia Fowler: Do you think E-cadherin in erythroblasts interacts with E-cadherin in other erythroblasts, or in other cell types? Posted onMarch 6, 2021 6:23 pm
Great session! FrancescaPosted onMarch 6, 2021 6:00 pm
Last question is a good title for a long talkPosted onMarch 6, 2021 5:58 pm
but inhibition of protein synthesis by phosphorylated eIF2a occurs in early erythroblasts before the massive globin synthesis. So I think that contributes to overall inhibition of erythropoiesis Posted onMarch 6, 2021 5:57 pm
Clara puoi ricordare the poster walk e le 2 meet the expert sessions graziePosted onMarch 6, 2021 5:57 pm
To Martina Great talk, thanks. It is known that Iron could also be a growth factor for tumor cells I am wondering whether in your model, tumor cells can take up iron nanoparticles or if they accumulate specifically in macrophages.Posted onMarch 6, 2021 5:56 pm
Thank you Clara and thank you all the speakers. Excellent session NicaPosted onMarch 6, 2021 5:55 pm
It was Ella :) Posted onMarch 6, 2021 5:53 pm
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and is reduced protein synthesis then the cause of small ery's Posted onMarch 6, 2021 5:51 pm
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achillePosted onMarch 6, 2021 5:51 pm
What is the role for low overall protein production by HRI-mediated phosphorylation of eIF2a 'marieke von Lindern Posted onMarch 6, 2021 5:51 pm
why DMT1 cells deficient a re so little Posted onMarch 6, 2021 5:51 pm
Great job iron ladies, this was a wonderful session!Posted onMarch 6, 2021 5:48 pm
not a question, but my congratulations to all the speakers of this session. Patricia Aguilar Martinez (France)Posted onMarch 6, 2021 5:47 pm
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clara le domande erano le mie: achille Posted onMarch 6, 2021 5:46 pm
To Porto: Do you have limitation on using EV iron in some microcytic anemia or in inflammatory anemias? Posted onMarch 6, 2021 5:45 pm
How is the KER drug different from other activin trap drugs?Posted onMarch 6, 2021 5:42 pm
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For Dr. Porto. Very nice data and presentation. Not sure if I understood the phenotype of the thalassemic patient. I think you mentioned he was transfusion dependent but then he benefit from phlebotomy. How did he manage the anemia? Thanks Stefano Rivella-Philly-USPosted onMarch 6, 2021 5:39 pm
To Graca : atypical for you means that is not monogenic ? or is related to what we know in the more common casePosted onMarch 6, 2021 5:37 pm
For Dr. Muckenthaler. Great talk. Can you polarized M2 to M1macrophages using specific cytokines instead of iron? Stefano Rivella-Philly-USAPosted onMarch 6, 2021 5:36 pm
For Laura Silvestri: could you comment on the role of monoferric vs diferric transferric in regulating hepcidin, and how the ratio of monoferric vs diferric transferrin may change with stimulation of erythropoieisis?Posted onMarch 6, 2021 5:36 pm
For Dr. Feigenson: sotatercept (another activin II ligand trap) did not show very promising results. do you expect better results for your molecule? in which aspects is it different from luspatercept and sotatercept? Also, are you planning studies on thalassemias? thanks a lotPosted onMarch 6, 2021 5:35 pm
To Dr. Feigenson: How does the KER drug regulate erythropoietin production?Posted onMarch 6, 2021 5:35 pm
For Dr. Feigenson. Exciting data How Ker-050 is different from Luspatercept? Any insight in the target molecules? Thanks you. Stefano Rivella-Philadelphia-USA To all: wonderful sessionPosted onMarch 6, 2021 5:34 pm
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To Marina: Did you explore a thalassemia model? Antonio PigaPosted onMarch 6, 2021 5:34 pm
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For Prof. SIlvestri: great presentation, very inspiring! my question is: you showed that circulating iron decreases EPO production in the kidney via IRP1. what do you think is the biological significance of this mechanism? and how is it linked to ineffective erythropoiesis, where on the contrary persistent anemia causes increased levels of EPO, even in presence of iron overload? ThanksPosted onMarch 6, 2021 5:22 pm
To Dr Muckenthaler. Thank you for this talk. Tumor immune microenvironment allows to classify solid tumors and their prognosis depending on the presence of inflammatory M2 macrophages or a more predominant non-inflammatory invasion by lymphocytes. Did you have the opportunity to investigate whether injection of iron nanoparticles to non inflammatory tumors induced tumor cell apoptosis?Posted onMarch 6, 2021 5:18 pm
iron iOS important in all the cells of the body, however the quantity is very different from erythroid and nervous cells (for example) the mechanism of controls is always the same? Posted onMarch 6, 2021 4:41 pm
Maria you use epoetina or darbo in elderly patients? And High dose aswell? What do you think about the tromboembolic risco associated? Thank you.Posted onMarch 6, 2021 4:05 pm
************ Sympo *********Posted onMarch 6, 2021 3:39 pm
Can you explain the relation between NfK inhibition eryptosis and obesityPosted onMarch 6, 2021 3:07 pm
How does your gluco mechanism compare with the mechanisms described by Lodish and colleagues involving PPARa and other factors/pathwaysPosted onMarch 6, 2021 3:04 pm
Is mitapivat (piravute kinase activator) acting decreasing eryptosis, too? Posted onMarch 6, 2021 3:04 pm
Can you retrieve the cultured cells from the sponge model ? Posted onMarch 6, 2021 3:00 pm
Balduini: Are you using completely synthetic media? e.g. PVA to replace serum albumin?Posted onMarch 6, 2021 2:59 pm
Have you compared the proteomes from early RBCs ans RBCs going erypthosis ? F. Baklouti, Lyon, FrancePosted onMarch 6, 2021 2:57 pm
setting a 3d BM model from a patient affected by a hematological hereditary disorder could you manipulateor treat the cells to correct the disorderPosted onMarch 6, 2021 2:55 pm
Are the platelets produced in the BM model viable and functional; could your model be use to produce erythrocytes and platelets?Posted onMarch 6, 2021 2:48 pm
Thank you, Alessandra, for this talk. This a very interesting and exciting approach. I have a technical question: how do you control molecular gradients in time and space? Is flow system enough efficient? Mariano (Paris) Posted onMarch 6, 2021 2:45 pm
in DBA, there is a cell cycle arrest in Go/G1 with less cells in phase S, do you think it may interferfer in the response of CFU-e proliferation between cord blood versus peripheral blood in DBA patient. difficult to collect cord blood from DBA patient and then follow the culture from the peripheral blood from the same patient. but it will be interesting to look if there is also some differences in that case. Posted onMarch 6, 2021 2:35 pm
Thank you for your talk. What are the mechanisms involved in cytokines uptake inside RBC? Are this mechanisms specific of RBC? What is the final fate of cytokines inside RBC? Mariano (Paris)Posted onMarch 6, 2021 2:29 pm
very nice talk lionel. congrats. my question: when we were doing only culture in methylcellulose with or without DXM in CD34+ from peripheral blood from DBA patients, some patients with good DXM response in vivo responded as well in vitro to DXM with increased proliferation but some don't suggesting no correlation between in vivo and vitro response to DXM in patient (reverse was true as well, bad response in vitro and good response in vivo). have you noticed as well some discrepancies in your experience like that with p57 evaluation? and perhaps it will be interesting to find some patients with this kind of discrepancy and look at p57 and so to identify why this lack of correlation between in vivo in vitro in some cases (new pathways? exogenous or endogenous modifiers?)? all the best and thanks for the teasing for tomorrowPosted onMarch 6, 2021 2:28 pm
Lionel, great talk. What is the mechanism of p57 induction by dex in adult erythroid progenitors. Michaela FontenayPosted onMarch 6, 2021 2:18 pm
Thanks for this talk. Do you know whether covid-19 patients treated with dex have any complication related to progenitors’ proliferation? Specially these in intensive care. Posted onMarch 6, 2021 2:17 pm
Jonas Schulte-Schrepping, Bonn: Thanks for taking up my rather long questions and answering them in so much detail. Super interesting and fascinating insights!Posted onMarch 6, 2021 1:51 pm
Sorry, I forgot to add my name and country to the questions to Prof Sankaran before: Jonas Schulte-Schrepping, Bonn Germany, Speaker in Session 1 yesterday. I have another Question to Prof. Sankaran with regard to the multiome solution from 10X. Did you observe the cells of one clone of terminally differentiated hematopoietic cells to be rather homogeneous in their transcriptome or spanning a broad range of phenotypes. And how were these observations reflected in the open chromatin profiles?Posted onMarch 6, 2021 1:44 pm
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Hi, I am Siyu from Oxford, here is my question for Dr. Sankaran: in the single cell ATAC-seq, you fixed the cells with 1% formaldehyde prior lysis and tagmentation to reduce contamination, I was wondering is there any difference in terms of results compared to standard scATAC-seq without the fixation step?Posted onMarch 6, 2021 1:32 pm
To Prof. Sankaran: This question might be rather unintuitive giving the high value of mitochondrial reads in your analyses. But, since mitochondrial reads in scRNA-seq often take up a lot of the generated reads and do not always contain relevant information given the given biological question and your extensive work on those reads, did you also find a way how to reduce those reads from scRNA-seq libraries to leave more space for more informative transcripts?Posted onMarch 6, 2021 1:23 pm
Did you already test your approach of lineage tracing/clonality analysis using the new combined scRNA+ATAC-seq methodology from 10X? This should allow to match phenotype and clonality even better. Posted onMarch 6, 2021 1:19 pm
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Laura Silvestri, Milan What is the mechanism of erythropoiesis improvement by vitamin B6 administration in R411H transgenic mice?Posted onMarch 6, 2021 12:48 pm
besides ineffective erythropoiesis is there a reduction of life span of siderocytes? Clara Camaschella - MilanoPosted onMarch 6, 2021 12:48 pm
********** Day 2 ********Posted onMarch 6, 2021 12:37 pm
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Are there any contraindications to the SARS-CoV-2 vaccine for patients with G6PD deficiency due to vaccine components?Posted onMarch 6, 2021 11:52 am
Are there any contraindications to the SARS-CoV-2 vaccine for patients with G6PD deficiency due to vaccine components?Posted onMarch 6, 2021 11:50 am
Posted onMarch 6, 2021 8:53 am
Thank you Ismael Posted onMarch 5, 2021 6:39 pm
Thanks for the talk by Ismael, can I ask is there any possibility to change the codon composition to increase the translation rate for RPS14 deficient patients? Have you tried this? Thanks. Posted onMarch 5, 2021 6:28 pm
Is it possible to decrease ROS to improve ineffective erythropoiesis?Posted onMarch 5, 2021 6:27 pm
Marc, Are there unique markers for the mesenchymal cells that talk to erythroid cells vs. more general mesenchymal cells in the bone marrow? Emery Bresnick, WIPosted onMarch 5, 2021 6:26 pm
Ismael, Nice presentation. Does the GATA1 translational downregulation impact a small fraction of GATA1-regulated genes/proteins or a highly restricted cohort? emery bresnick WIPosted onMarch 5, 2021 6:24 pm
Why MDS LOW RISK AS REFRACTORY ANEMIA OR RARS WHICH CHARACTERISED BY INEFFECTIVE ERYTHROPOIESIS DO NOT PRESENT SO COMMON EXTRAMEDULARY ERYTHROPOIESIS AS THALASSEMIA INDERMEDIA? Posted onMarch 5, 2021 5:58 pm
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What about the controversy of the data of GDF11 erythroid KO mice that respond as wild type to action ligand trap? Clara Camaschella, MilanoPosted onMarch 5, 2021 5:56 pm
How do you explain the effect of Luspatercept in beta0/beta0 thalassemia patients?Posted onMarch 5, 2021 5:53 pm
Laura Silvestri, Milano In vivo studies exclude GDF11 as a player in ineffective erythropoiesis and the ligand sequestered by ACE molecules. What about GDF8? There is any role for this ligand in ineffective erythropoiesis?Posted onMarch 5, 2021 5:47 pm
******** Session 3 **********Posted onMarch 5, 2021 5:34 pm
Do SF3B1 mutations affect splicing and translation of key erythroid transcription factors?Posted onMarch 5, 2021 5:34 pm
Hi all, what do you think about the recently published paper 'Human erythroid progenitors are directly infected by SARS-CoV-2: implications for emerging erythropoiesis in severe COVID19 patients' on Stem Cell Reports about a specific population of herythrocytes being more susceptible for infection? Thanks.Posted onMarch 5, 2021 5:03 pm
hola buen dia soy el dr meza de hetaologia en mexico, seguimos encontrando linfopenia severa con neutrofilia como una de las cosas mas frecuentes en paciente en estado critico en mexico con esteroides a dosis bajas dexametasona 6-8 mg cada 24 hrs , ¿creen que tenga un beneficio aumentar la dosi de esteroide a 24 mgs ) o pulos de metilpredinoslona 500 mgs cada 24 hrs ?Posted onMarch 5, 2021 4:55 pm
For Prof. Tolosano and Motta: could you comment about any possible connection among your data, specifically is there a role of heme methabolism in explaining covid response observed among beta-thalassemia patients? And what about SCD? ThanksPosted onMarch 5, 2021 4:44 pm
May I miss this. Italy Blood bank may have done routinarlly any PCR Covid Test for assymptomatic blood donors ?Posted onMarch 5, 2021 4:43 pm
Posted onMarch 5, 2021 4:43 pm
Please can we get this lecture as PowerPoint Posted onMarch 5, 2021 4:41 pm
Marieke von Lindern : Covid patients complain about prolonged fatigue (also after relatively mild symptoms) is this linked to Hb and hemoglobin?Posted onMarch 5, 2021 4:03 pm
Posted onMarch 5, 2021 4:02 pm
to Hal Drakesmith by Marieke von Lindern (Sanquin, Amsterdam): Does the axes of IL6-hepcidin-low iron regulate the proliferation of the lymphoid cells of the adaptive immune in general (suggested by the model on your conclusion slide), or does iron availability much more affect the balance in activity between T effector cells and T regulatory cells ? Posted onMarch 5, 2021 3:53 pm
Laura Silvestri, Milan Fe/S cluster and heme biosynthesis are two processes closely interconnected. Fe/s clusters are prosthetic groups for enzymes of the respiratory chain and krebs cycle. Is the Fe/S cluster machinery affected by FLVCR1a silencing or overexpression. Do you think this biosynthetic pathway can partially explain the cell phenotype you described?Posted onMarch 5, 2021 3:44 pm
A pediatric disease named IRIDA causes hypoferremia due to constitutively high hepcidin levels. I was wondering whether an impaired immune response after vaccination has been described for these patients.Posted onMarch 5, 2021 3:34 pm
Clara Camaschella, Milano As for anti COVId vaccine is there any evidence of different response of iron deficient individuals?Posted onMarch 5, 2021 3:32 pm
Are the presentations recorded so that I can look back?Posted onMarch 5, 2021 3:05 pm
************* Session 2 ********Posted onMarch 5, 2021 3:01 pm
Posted onMarch 5, 2021 1:58 pm
What's the difference between these immature Neutrophils and MDSCs?Posted onMarch 5, 2021 1:52 pm
What is the difference between the immature neutrophils and MDSCs? Posted onMarch 5, 2021 1:47 pm
Petros Papadopoulos/Madrid Was there any platelet gene expression signature in the COVID patients in whole blood RNAseq?Posted onMarch 5, 2021 1:47 pm
Verdier Frédérique (france) for Dr Nai: A question about the recovery of anemia in beta thalassemia in tfr2BM KO mice. Is it possible that the absence of TFR2 in erythroblasts inhibit erythroid maturation, that consequently induce stress erythropoiesis and mobilization of iron in excess in this pathology ? Posted onMarch 5, 2021 1:42 pm
globin editing speaker: Can you discuss immunogenicity considerations with the in vivo editing strategy and if relevant, are unique measures taken to mitigate immune-related issues? emery bresnick, WIPosted onMarch 5, 2021 1:39 pm
TO Dr. Schrepping: is there any therapeutic approach that may counteract COVID-related emergency hemopoiesis? Posted onMarch 5, 2021 1:35 pm
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for CasanovaPosted onMarch 5, 2021 1:32 pm
Considering the battery of functionally significant genetic variants demonstrated to cause or contribute to primary immunodeficiency disease, did the Covid patient sequencing reveal the majority of all of these variants or were certain variants conspicuously absent emery bresnick, WIPosted onMarch 5, 2021 1:32 pm
For Ms Georgakopoulou: Thank you for the presentation. Have you checked if there is any translocation in your edited HSCs, as you target more than 1 target? Have you also thought to replace the Cas9 with base editors in order to avoid any such event, if present? Thank you, Panagiotis Antoniou, IMAGINE Institute, Paris, FrancePosted onMarch 5, 2021 1:28 pm
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Laura Silvestri, Milan What is the frequency of inborn errors in Interferon-gamma genes in the general population? Posted onMarch 5, 2021 12:48 pm
123Posted onMarch 5, 2021 8:12 am
TestsssPosted onMarch 5, 2021 8:11 am
********Day 1********Posted onMarch 5, 2021 7:58 am
test 123Posted onMarch 3, 2021 7:28 pm