Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial-Reference-Cited by-同舟云学术

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

Published:2022-01 Issue:1 Volume:28 Page:81-88
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Magrin Elisa,Semeraro Michaela,Hebert Nicolas^ORCID,Joseph Laure,Magnani Alessandra,Chalumeau Anne,Gabrion Aurélie^ORCID,Roudaut Cécile,Marouene Jouda,Lefrere Francois,Diana Jean-Sebastien^ORCID,Denis Adeline,Neven Bénédicte,Funck-Brentano Isabelle,Negre Olivier,Renolleau Sylvain^ORCID,Brousse Valentine^ORCID,Kiger Laurent,Touzot Fabien,Poirot Catherine,Bourget Philippe,El Nemer Wassim^ORCID,Blanche Stéphane,Tréluyer Jean-Marc,Asmal Mohammed,Walls Courtney,Beuzard Yves,Schmidt Manfred,Hacein-Bey-Abina Salima,Asnafi Vahid,Guichard Isabelle,Poirée Maryline,Monpoux Fabrice,Touraine Philippe,Brouzes Chantal,de Montalembert Mariane,Payen Emmanuel^ORCID,Six Emmanuelle^ORCID,Ribeil Jean-Antoine,Miccio Annarita^ORCID,Bartolucci Pablo,Leboulch Philippe^ORCID,Cavazzana Marina^ORCID

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

https://www.nature.com/articles/s41591-021-01650-w.pdf

Reference38 articles.

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2. Pasricha, S. R. & Drakesmith, H. Hemoglobinopathies in the fetal position. N. Engl. J. Med. 379, 1675–1677 (2018).

3. Shah, F. T., Sayani, F., Trompeter, S., Drasar, E. & Piga, A. Challenges of blood transfusions in β-thalassemia. Blood Rev. 37, 100588 (2019).

4. Cappellini, M. D. et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N. Engl. J. Med. 382, 1219–1231 (2020).

5. Niihara, Y. et al. A phase 3 trial of ʟ-glutamine in sickle cell disease. N. Engl. J. Med. 379, 226–235 (2018).

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