Hematopoietic Stem-Cell Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, David A. Williams, Satiro De Oliveira et al.|New England Journal of Medicine|2017Cited by 592
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trialPerry B. Shieh, Salvador Rico, Nancy L. Kuntz et al.|The Lancet Neurology|2023Cited by 120