Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. However, insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. (Funded by Bluebird Bio; ALD-102 and LTF-304 ClinicalTrials.gov numbers NCT01896102 and NCT02698579, respectively.).
Authors
- Daniel J. Loes (ORCID: https://orcid.org/0000-0001-7661-3442)
- Patrick Aubourg
- Jean‐Hugues Dalle (ORCID: https://orcid.org/0000-0002-8406-0793)
- Elizabeth McNeil (ORCID: https://orcid.org/0000-0003-3439-3722)
- Adrian J. Thrasher (ORCID: https://orcid.org/0000-0002-6097-6115)
- Himal L. Thakar
- Shaun A. Hussain (ORCID: https://orcid.org/0000-0001-6947-8852)
- Patricia L. Musolino (ORCID: https://orcid.org/0000-0001-8724-5649)
- Jörn‐Sven Kühl (ORCID: https://orcid.org/0000-0002-1739-1090)
- Satiro De Oliveira (ORCID: https://orcid.org/0000-0002-8181-7316)
- Troy C. Lund (ORCID: https://orcid.org/0000-0001-9174-8919)
- Florian Eichler (ORCID: https://orcid.org/0000-0001-6836-1089)
- Juliana Folloni Fernandes (ORCID: https://orcid.org/0000-0002-5764-1621)
- Nicholas Smith (ORCID: https://orcid.org/0000-0002-0324-3054)
- Paul J. Orchard (ORCID: https://orcid.org/0000-0001-9426-1292)
- Caroline Sevin (ORCID: https://orcid.org/0000-0002-5856-068X)
- David A. Williams (ORCID: https://orcid.org/0000-0001-7057-6863)
- Laura A. Demopoulos
- Raman Sankar (ORCID: https://orcid.org/0000-0002-5609-0362)
- Uwe Platzbecker (ORCID: https://orcid.org/0000-0003-1863-3239)
- Ashish O. Gupta
- Paul Gissen (ORCID: https://orcid.org/0000-0002-9712-6122)
- Andrew C. Dietz (ORCID: https://orcid.org/0000-0003-2810-2466)
- Gerald Downey
- Christine Duncan (ORCID: https://orcid.org/0000-0002-2090-4897)
- Hernán Amartino
Institutions
- Inserm (FR)
- Université Paris-Saclay (FR)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
- University College London (GB)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2024-10-09
- DOI
- https://doi.org/10.1056/nejmoa2400442
- Citations
- 61
- Primary Topic
- Peroxisome Proliferator-Activated Receptors
- Type
- article
- Field-Weighted Citation Impact
- 14.26
Funders
- Massachusetts General Hospital
- University of Minnesota
- bluebird bio
- National Institute for Health and Care Research
- University College London
- Institut National de la Santé et de la Recherche Médicale
- Universidade de São Paulo
- Université Paris-Saclay
- Assistance publique-Hôpitaux de Paris
- Université de Paris
- University of California, Los Angeles