Interim Results from a Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy With Lentiviral Vector (Lenti-D) for Childhood Cerebral Adrenoleukodystrophy (PL02.002)
Year: 2016
Type: article
Study design: Clinical Trial
Source: Neurology
Authors: Florian Eichler, Christine Duncan, Adrian Thrasher, Ami Shah, Andre Baruchel, Asif Paker, Donald Kohn, Drago Bratkovic, Esther Shamir, Gerald Raymond, H. Bobby Gaspar, Harnan Amartino, Jean-Hugues Dalle, Jerome Larghero, Nicholas Smith, Patricia Musolino, Paul Gissen, Paul Orchard, Raman Sankar, Satiro De Oliveira, Tara O'Meara, Troy Lund, Weston Miller, Patrick Aubourg, David Williams
Institutions: Massachusetts General Hospital, Dana-Farber Cancer Institute, University College London, Hôpital Saint-Louis, Bluebird Bio (United States), University of California, Los Angeles, University of Minnesota, Hôpital Robert-Debré, Women's and Children's Hospital, SA Health, Great Ormond Street Hospital, Université Paris-Sud, Boston Children's Hospital
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: closed
OpenAlex ID: W2508738895