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4. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. Frangoul H; Altshuler D; Cappellini MD; Chen YS; Domm J; Eustace BK; Foell J; de la Fuente J; Grupp S; Handgretinger R; Ho TW; Kattamis A; Kernytsky A; Lekstrom-Himes J; Li AM; Locatelli F; Mapara MY; de Montalembert M; Rondelli D; Sharma A; Sheth S; Soni S; Steinberg MH; Wall D; Yen A; Corbacioglu S N Engl J Med; 2021 Jan; 384(3):252-260. PubMed ID: 33283989 [TBL] [Abstract][Full Text] [Related]
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6. Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia. Thompson AA; Walters MC; Kwiatkowski J; Rasko JEJ; Ribeil JA; Hongeng S; Magrin E; Schiller GJ; Payen E; Semeraro M; Moshous D; Lefrere F; Puy H; Bourget P; Magnani A; Caccavelli L; Diana JS; Suarez F; Monpoux F; Brousse V; Poirot C; Brouzes C; Meritet JF; Pondarré C; Beuzard Y; Chrétien S; Lefebvre T; Teachey DT; Anurathapan U; Ho PJ; von Kalle C; Kletzel M; Vichinsky E; Soni S; Veres G; Negre O; Ross RW; Davidson D; Petrusich A; Sandler L; Asmal M; Hermine O; De Montalembert M; Hacein-Bey-Abina S; Blanche S; Leboulch P; Cavazzana M N Engl J Med; 2018 Apr; 378(16):1479-1493. PubMed ID: 29669226 [TBL] [Abstract][Full Text] [Related]
7. Exagamglogene Autotemcel: First Approval. Hoy SM Mol Diagn Ther; 2024 Mar; 28(2):133-139. PubMed ID: 38228954 [TBL] [Abstract][Full Text] [Related]
8. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia. Ye L; Wang J; Tan Y; Beyer AI; Xie F; Muench MO; Kan YW Proc Natl Acad Sci U S A; 2016 Sep; 113(38):10661-5. PubMed ID: 27601644 [TBL] [Abstract][Full Text] [Related]
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19. Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience. Zaidman I; Rowe JM; Khalil A; Ben-Arush M; Elhasid R Biol Blood Marrow Transplant; 2016 Jun; 22(6):1043-1048. PubMed ID: 27016193 [TBL] [Abstract][Full Text] [Related]
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