Publication
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Florian Eichler, Christine N. Duncan, Patricia L. Musolino, Troy C. Lund, Ashish O. Gupta, Satiro De Oliveira, Adrian J. Thrasher, Patrick Aubourg, Jörn-Sven Kühl, Daniel J. Loes, Hernan Amartino, Nicholas Smith, Juliana Folloni Fernandes, Caroline Sevin, Raman Sankar, Shaun A. Hussain, Paul Gissen, Jean-Hugues Dalle, Uwe Platzbecker, Gerald F. Downey, Elizabeth McNeil, Laura Demopoulos, Andrew C. Dietz, Himal L. Thakar, Paul J. Orchard, David A. Williams
The New England Journal of Medicine and Surgery and the Collateral Branches of Science, October 2024, New England Journal of Medicine (NEJM/MMS)
DOI: 10.1056/nejmoa2400442