Efficacy and Safety of Gene Therapy for β-Thalassemia

Affiliations

¹ From the Division of Innovative Therapies, Infectious Diseases Models for Innovative Therapies Research Center and Unité Mixte de Recherche-1184, French Alternative Energies and Atomic Energy Commission Fontenay-aux-Roses, INSERM, Université Paris-Saclay, Fontenay-aux-Roses, France.

Editorial

Emmanuel Payen. N Engl J Med. 2022.

. 2022 Feb 3;386(5):488-490.

doi: 10.1056/NEJMe2118580.

¹ From the Division of Innovative Therapies, Infectious Diseases Models for Innovative Therapies Research Center and Unité Mixte de Recherche-1184, French Alternative Energies and Atomic Energy Commission Fontenay-aux-Roses, INSERM, Université Paris-Saclay, Fontenay-aux-Roses, France.

No abstract available

Comment on

Betibeglogene Autotemcel Gene Therapy for Non-β⁰/β⁰ Genotype β-Thalassemia.
Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Locatelli F, et al. N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11. N Engl J Med. 2022. PMID: 34891223 Clinical Trial.