Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
These long-term findings in a large patient cohort show the sustained clinical efficacy and safety of autologous CD34+ hematopoietic stem-cell lentiviral gene therapy for ADA-SCID, indicating that it is a curative treatment. (Funded by the National Heart, Lung, and Blood Institute and others; ClinicalTrials.gov number, NCT04049084.).
Authors
- Augustine Fernandes
- Claire Booth (ORCID: https://orcid.org/0000-0002-2626-5037)
- Harry L. Malech (ORCID: https://orcid.org/0000-0001-5874-5775)
- Adrian J. Thrasher (ORCID: https://orcid.org/0000-0002-6097-6115)
- Konstantinos Vazouras (ORCID: https://orcid.org/0000-0002-5278-0083)
- Lana Mhaldien
- Katelyn E. Masiuk (ORCID: https://orcid.org/0000-0001-5372-4166)
- Elena Alvarez Mediavilla
- Satiro De Oliveira (ORCID: https://orcid.org/0000-0002-8181-7316)
- Havinder Hara
- Grainne O’Toole
- Beatriz Campo Fernandez
- Donald B. Kohn (ORCID: https://orcid.org/0000-0003-1840-6087)
- Theodore B. Moore (ORCID: https://orcid.org/0000-0002-7909-610X)
- Stuart Adams (ORCID: https://orcid.org/0000-0002-2650-2848)
- Jinhua Xu‐Bayford
- Danilo Pellin (ORCID: https://orcid.org/0000-0002-2647-0508)
- Elizabeth Garabedian
- Kimberly Gilmour (ORCID: https://orcid.org/0000-0003-0285-3958)
- Suk See De Ravin (ORCID: https://orcid.org/0000-0002-9800-774X)
- Kenneth Cornetta (ORCID: https://orcid.org/0000-0003-1415-3668)
- Tsai‐Yu Lin (ORCID: https://orcid.org/0000-0002-8076-1584)
- Sohini Roy
- Thao Thi Dang
- James E. Arnold
- H. Bobby Gaspar
- Dayna Terrazas
- Razzaque Ahmed
- Michael S. Hershfield
- Jack L. Reid (ORCID: https://orcid.org/0009-0005-8080-8302)
- Beatrice Curio-Penny
Institutions
- National Institutes of Health (US)
- University of California, Los Angeles (US)
- Great Ormond Street Hospital (GB)
- Great Ormond Street Hospital for Children NHS Foundation Trust (GB)
- National Human Genome Research Institute (US)
- Duke Medical Center (US)
- National Institute of Allergy and Infectious Diseases (US)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
- Indiana University School of Medicine
- University College London (GB)
- Indiana University – Purdue University Indianapolis (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-10-15
- DOI
- https://doi.org/10.1056/nejmoa2502754
- Citations
- 7
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 12.72