A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

2014404 citationsJournal Articlebronze Open Access

Authors

Salima Hacein‐Bey‐Abina · Assistance Publique – Hôpitaux de Paris

Sung‐Yun Pai · Harvard University

H. Bobby Gaspar · University College London

Myriam Armant · Harvard University

Charles C. Berry · Cincinnati Children's Hospital Medical Center

Stéphane Blanche · Université Paris Cité

Jack Bleesing

Abstract

This modified γ-retrovirus vector was found to retain efficacy in the treatment of SCID-X1. The long-term effect of this therapy on leukemogenesis remains unknown. (Funded by the National Institutes of Health and others; ClinicalTrials.gov numbers, NCT01410019, NCT01175239, and NCT01129544.).

Topics & Keywords

Virus-based gene therapy research CAR-T cell therapy research CRISPR and Genetic Engineering

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 371, Issue 15, pp. 1407-1417

DOI: 10.1056/nejmoa1404588

Field-Weighted Citation Impact: 38.03