Should patients with haemophilia receive gene therapy?

TY - JOUR

T1 - Should patients with haemophilia receive gene therapy?

AU - VandenDriessche, Thierry

AU - Tatsumi, Kohei

AU - Shima, Midori

N1 - Funding Information: TVD declares funding from Pfizer and Catalyst Biosciences; speaker fees from Takeda, Pfizer, and BioMarin; research grants from European Union Horizon 2020 UPGRADE project and Vrije Universiteit Brussel IOF GEAR; and patents on vector designs for haemophilia gene therapy. TVD thanks Glenn Pierce, David Lillicrap, and Alok Srivastava for helpful comments. MS declares grants from Chugai, CSL-Behring, Takeda; consulting fess from Chugai, Fujimoto, and UniQure; and speaker fees from Chugai, Roche, Novo Nordisk, CSL-Behring, Sanofi, Bayer and Fujimoto. KT declares grants or research support from Grants-in-Aid for Scientific Research (KAKENHI), Japan Blood Products Organization, The Mother and Child Health Foundation, and Novo Nordisk. Copyright: Copyright 2023 Elsevier B.V., All rights reserved.

PY - 2022/10

Y1 - 2022/10

KW - Genetic Therapy

KW - Hemophilia A/genetics

KW - Humans

UR - http://www.scopus.com/inward/record.url?scp=85138810107&partnerID=8YFLogxK

U2 - 10.1016/S2352-3026(22)00300-3

DO - 10.1016/S2352-3026(22)00300-3

M3 - Comment/debate

C2 - 36174639

SN - 2352-3026

VL - 9

SP - e722-e723

JO - The Lancet. Haematology

JF - The Lancet. Haematology

IS - 10

ER -