Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

More than 30 years ago, the first transplantation with autologous hematopoietic stem cells modified with integrated murine retroviral vectors carrying potentially therapeutic genes was performed in patients with serious human disorders. The past decade has been marked by an acceleration in the publication of data from clinical trials and in…

​   The New England Journal of Medicine: Search Results in Pediatrics