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About 379 results

ALLMedicine™ Inherited Bone Marrow Failure Syndromes Center

Research & Reviews  187 results

Implications of hematopoietic stem cells heterogeneity for gene therapies.
https://doi.org/10.1038/s41434-021-00229-x 10.1182/blood-2002-09-2800 10.1126/science.270.5235.475 10.1056/NEJMoa1000164 10.1126/science.1233151 10.1056/NEJMoa1705342 10.1056/NEJMoa1609677 10.1182/blood.V74.2.876.876 10.1038/bmt.2015.6 10.1038/s41422-019-0228-6 10.1038/s41556-018-0121-4 10.1016/j.stem.2013.05.014 10.1016/j.cell.2008.10.048 10.1016/j.stem.2014.07.005 10.1016/j.stem.2015.05.003 10.1111/j.1365-2141.1999.01337.x 10.1016/j.omtm.2017.03.003 10.1038/s41573-019-0020-9 10.1016/j.stem.2017.10.010 10.1182/blood-2010-08-302109 10.1038/sj.leu.2401603 10.1038/sj.bmt.1704758 10.1016/S0140-6736(84)92564-9 10.1016/j.stem.2018.04.003 10.1007/s00018-016-2306-y 10.1038/s41556-019-0444-9 10.1182/blood-2003-10-3448 10.1073/pnas.1116110108 10.1182/blood-2014-12-570200 10.1172/JCI103949 10.1038/197452a0 10.1084/jem.127.3.455 10.1182/blood-2008-08-078220 10.2353/ajpath.2006.060312 10.1016/j.cell.2017.04.018 10.1016/j.stem.2015.01.017 10.1038/sj.bmt.1703566 10.1038/mt.2014.173 10.1242/dev.114231 10.1002/stem.1661 10.1073/pnas.94.10.5320 10.1007/978-1-4614-5894-4_7 10.1038/s41467-019-12726-0 10.1111/bjh.13362 10.1111/trf.14541 10.1016/j.stem.2013.05.025 10.1038/s41467-018-06442-4 10.1016/j.stem.2014.10.020 10.1182/blood-2016-03-704668 10.1016/j.stem.2012.02.007 10.1016/j.celrep.2019.05.028 10.1182/blood.2020006229 10.1016/j.stem.2007.10.020 10.1038/nature14131 10.1038/ncomms15099 10.1038/bmt.2016.88 10.1016/j.bbmt.2017.05.003 10.1016/j.bbmt.2009.10.027 10.1016/j.cyto.2017.09.001 10.1038/nature13420 10.1182/blood.2019002350 10.1038/nm.3647 10.1038/s41556-019-0439-6 10.1038/srep37827 10.1097/MOH.0000000000000347 10.1038/ncomms9040 10.1016/j.stem.2010.02.002 10.1073/pnas.1801480116 10.1111/j.1423-0410.2012.01632.x 10.1038/leu.2011.197 10.1038/bmt.2014.39 10.1016/j.cell.2017.11.003 10.3324/haematol.2012.077040 10.1182/blood-2015-03-629618 10.1182/blood-2011-12-376749 10.1016/j.cell.2013.08.007 10.1016/j.stem.2013.12.012 10.1038/nature12013 10.1016/S0140-6736(10)61029-X 10.1371/journal.pmed.1001484 10.1056/NEJM200006223422502 10.2471/BLT.06.036673 10.1016/S0140-6736(17)31822-6 10.1182/blood.V128.22.1176.1176 10.1182/blood-2010-09-255679 10.1126/science.1165409 10.1073/pnas.0711566105 10.1056/NEJMra1510865 10.1056/NEJMoa2031054 10.1038/mtna.2015.37 10.1073/pnas.1714640114 10.1016/j.celrep.2016.08.064 10.1186/s13059-015-0739-5 10.1016/j.omtm.2016.12.009 10.1172/JCI87885 10.1016/j.omtm.2020.03.015 10.1056/NEJMp068209 10.1097/MOH.0b013e3282f30448 10.1038/nri2724 10.1182/blood-2008-12-115253 10.1182/blood-2005-03-1219 10.1056/NEJMoa1003548 10.1126/scitranslmed.3007280 10.1016/j.gene.2013.03.092 10.1038/s41591-018-0195-3 10.1016/0092-8674(93)90167-O 10.1146/annurev.immunol.14.1.179 10.1016/S0022-1759(00)00136-8 10.1086/302519 10.1146/annurev.immunol.19.1.331 10.3389/fmicb.2018.02940 10.1126/science.273.5283.1856 10.1016/S0092-8674(00)80110-5 10.1016/j.atg.2013.05.004 10.1056/NEJMoa0802905 10.1016/S2352-3018(20)30069-2 10.1056/NEJMoa1300662 10.1056/NEJMoa1817426 10.1016/j.stemcr.2017.02.010 10.1073/pnas.1000834107 10.1038/s41375-020-0982-y 10.1084/jem.20041385
Gene Therapy; Epah J, Schäfer R

Feb 16th, 2021 - Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allo...

Hematopoietic stem cell transplantation for inherited bone marrow failure syndromes: al...
https://doi.org/10.1080/16078454.2021.1876393
Hematology (Amsterdam, Netherlands); Lu Y, Xiong M et. al.

Jan 25th, 2021 - Objective: The outcomes of alternative donor hematopoietic stem cell transplantation (HSCT) with unmanipulated grafts for Inherited bone marrow failure syndromes (IBMFS) are discouraging. Our study is to demonstrate that IBMFS with disease-specifi...

Multiorgan failure with abnormal receptor metabolism in mice mimicking Samd9/9L syndromes.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7880413
The Journal of Clinical Investigation; Nagamachi A, Kanai A et. al.

Dec 29th, 2020 - Autosomal dominant sterile α motif domain containing 9 (Samd9) and Samd9L (Samd9/9L) syndromes are a large subgroup of currently established inherited bone marrow failure syndromes that includes myelodysplasia, infection, growth restriction, adren...

Rasa3 regulates stage-specific cell cycle progression in murine erythropoiesis.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7856249
Blood Cells, Molecules & Diseases; Brindley EC, Papoin J et. al.

Dec 19th, 2020 - Inherited bone marrow failure syndromes (IBMFS) are heterogeneous disorders characterized by dysregulated hematopoiesis in various lineages, developmental anomalies, and predisposition to malignancy. The scat (severe combined anemia and thrombocyt...

see more →

Guidelines  2 results

Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherite...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5565711
Biology of Blood and Marrow Transplantation : Journal of ... Dietz AC, Savage SA et. al.

May 23rd, 2017 - Patients with inherited bone marrow failure syndromes (IBMFS), such as Fanconi anemia (FA), dyskeratosis congenita (DC), or Diamond Blackfan anemia (DBA), can have hematologic manifestations cured through hematopoietic cell transplantation (HCT). ...

Recommendations on hematopoietic stem cell transplantation for inherited bone marrow fa...
https://doi.org/10.1038/bmt.2015.117
Bone Marrow Transplantation; Peffault de Latour R, Peters C et. al.

Jun 8th, 2015 - Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated w...

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Clinicaltrials.gov  189 results

Implications of hematopoietic stem cells heterogeneity for gene therapies.
https://doi.org/10.1038/s41434-021-00229-x 10.1182/blood-2002-09-2800 10.1126/science.270.5235.475 10.1056/NEJMoa1000164 10.1126/science.1233151 10.1056/NEJMoa1705342 10.1056/NEJMoa1609677 10.1182/blood.V74.2.876.876 10.1038/bmt.2015.6 10.1038/s41422-019-0228-6 10.1038/s41556-018-0121-4 10.1016/j.stem.2013.05.014 10.1016/j.cell.2008.10.048 10.1016/j.stem.2014.07.005 10.1016/j.stem.2015.05.003 10.1111/j.1365-2141.1999.01337.x 10.1016/j.omtm.2017.03.003 10.1038/s41573-019-0020-9 10.1016/j.stem.2017.10.010 10.1182/blood-2010-08-302109 10.1038/sj.leu.2401603 10.1038/sj.bmt.1704758 10.1016/S0140-6736(84)92564-9 10.1016/j.stem.2018.04.003 10.1007/s00018-016-2306-y 10.1038/s41556-019-0444-9 10.1182/blood-2003-10-3448 10.1073/pnas.1116110108 10.1182/blood-2014-12-570200 10.1172/JCI103949 10.1038/197452a0 10.1084/jem.127.3.455 10.1182/blood-2008-08-078220 10.2353/ajpath.2006.060312 10.1016/j.cell.2017.04.018 10.1016/j.stem.2015.01.017 10.1038/sj.bmt.1703566 10.1038/mt.2014.173 10.1242/dev.114231 10.1002/stem.1661 10.1073/pnas.94.10.5320 10.1007/978-1-4614-5894-4_7 10.1038/s41467-019-12726-0 10.1111/bjh.13362 10.1111/trf.14541 10.1016/j.stem.2013.05.025 10.1038/s41467-018-06442-4 10.1016/j.stem.2014.10.020 10.1182/blood-2016-03-704668 10.1016/j.stem.2012.02.007 10.1016/j.celrep.2019.05.028 10.1182/blood.2020006229 10.1016/j.stem.2007.10.020 10.1038/nature14131 10.1038/ncomms15099 10.1038/bmt.2016.88 10.1016/j.bbmt.2017.05.003 10.1016/j.bbmt.2009.10.027 10.1016/j.cyto.2017.09.001 10.1038/nature13420 10.1182/blood.2019002350 10.1038/nm.3647 10.1038/s41556-019-0439-6 10.1038/srep37827 10.1097/MOH.0000000000000347 10.1038/ncomms9040 10.1016/j.stem.2010.02.002 10.1073/pnas.1801480116 10.1111/j.1423-0410.2012.01632.x 10.1038/leu.2011.197 10.1038/bmt.2014.39 10.1016/j.cell.2017.11.003 10.3324/haematol.2012.077040 10.1182/blood-2015-03-629618 10.1182/blood-2011-12-376749 10.1016/j.cell.2013.08.007 10.1016/j.stem.2013.12.012 10.1038/nature12013 10.1016/S0140-6736(10)61029-X 10.1371/journal.pmed.1001484 10.1056/NEJM200006223422502 10.2471/BLT.06.036673 10.1016/S0140-6736(17)31822-6 10.1182/blood.V128.22.1176.1176 10.1182/blood-2010-09-255679 10.1126/science.1165409 10.1073/pnas.0711566105 10.1056/NEJMra1510865 10.1056/NEJMoa2031054 10.1038/mtna.2015.37 10.1073/pnas.1714640114 10.1016/j.celrep.2016.08.064 10.1186/s13059-015-0739-5 10.1016/j.omtm.2016.12.009 10.1172/JCI87885 10.1016/j.omtm.2020.03.015 10.1056/NEJMp068209 10.1097/MOH.0b013e3282f30448 10.1038/nri2724 10.1182/blood-2008-12-115253 10.1182/blood-2005-03-1219 10.1056/NEJMoa1003548 10.1126/scitranslmed.3007280 10.1016/j.gene.2013.03.092 10.1038/s41591-018-0195-3 10.1016/0092-8674(93)90167-O 10.1146/annurev.immunol.14.1.179 10.1016/S0022-1759(00)00136-8 10.1086/302519 10.1146/annurev.immunol.19.1.331 10.3389/fmicb.2018.02940 10.1126/science.273.5283.1856 10.1016/S0092-8674(00)80110-5 10.1016/j.atg.2013.05.004 10.1056/NEJMoa0802905 10.1016/S2352-3018(20)30069-2 10.1056/NEJMoa1300662 10.1056/NEJMoa1817426 10.1016/j.stemcr.2017.02.010 10.1073/pnas.1000834107 10.1038/s41375-020-0982-y 10.1084/jem.20041385
Gene Therapy; Epah J, Schäfer R

Feb 16th, 2021 - Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allo...

Hematopoietic stem cell transplantation for inherited bone marrow failure syndromes: al...
https://doi.org/10.1080/16078454.2021.1876393
Hematology (Amsterdam, Netherlands); Lu Y, Xiong M et. al.

Jan 25th, 2021 - Objective: The outcomes of alternative donor hematopoietic stem cell transplantation (HSCT) with unmanipulated grafts for Inherited bone marrow failure syndromes (IBMFS) are discouraging. Our study is to demonstrate that IBMFS with disease-specifi...

Multiorgan failure with abnormal receptor metabolism in mice mimicking Samd9/9L syndromes.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7880413
The Journal of Clinical Investigation; Nagamachi A, Kanai A et. al.

Dec 29th, 2020 - Autosomal dominant sterile α motif domain containing 9 (Samd9) and Samd9L (Samd9/9L) syndromes are a large subgroup of currently established inherited bone marrow failure syndromes that includes myelodysplasia, infection, growth restriction, adren...

Rasa3 regulates stage-specific cell cycle progression in murine erythropoiesis.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7856249
Blood Cells, Molecules & Diseases; Brindley EC, Papoin J et. al.

Dec 19th, 2020 - Inherited bone marrow failure syndromes (IBMFS) are heterogeneous disorders characterized by dysregulated hematopoiesis in various lineages, developmental anomalies, and predisposition to malignancy. The scat (severe combined anemia and thrombocyt...

see more →

News  1 results

Topics in Pediatric Leukemia -- Fanconi's Anemia: New Insights
https://www.medscape.com/viewarticle/499947

Introduction The inherited bone marrow failure syndromes in children comprise a number of rare congenital disorders in which the bone marrow is unable to produce blood cells; this results in a deficiency of 1 or more cell lines. Studies on the pat...

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