Correcting the aberrant Fanconi anemia transcriptional program by gene therapy
Author:
Publisher
Ferrata Storti Foundation (Haematologica)
Subject
Hematology
Reference11 articles.
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2. Badra Fajardo N, Taraviras S, Lygerou Z. Fanconi anemia proteins and genome fragility: unraveling replication defects for cancer therapy. Trends Cancer. 2022; 8(6):467-481.
3. Bonfim C, Ribeiro L, Nichele S. Long-term survival, organ function, and malignancy after hematopoietic stem cell transplantation for Fanconi anemia. Biol Blood Marrow Transplant. 2016; 22(7):1257-1263.
4. Alter BP, Giri N, Savage SA, Rosenberg PS. Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up. Haematologica. 2018; 103(1):30-39.
5. Rio P, Navarro S, Wang W. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nat Med. 2019; 25(9):1396-1401.
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1. T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML;Bone Marrow Transplantation;2023-09-29
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