Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were free of major disability. Myelodysplastic syndrome...
Case 31-2024: A 37-Year-Old Man with Fever, Myalgia, Jaundice, and Respiratory Failure
A 37-year-old man was transferred to the hospital because of fever, myalgia, and hypoxemia. Evaluation revealed leukocytosis, acute kidney failure, and conjugated hyperbilirubinemia. A...
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Hematologic cancer (mainly myelodysplastic syndrome) developed in 7 of 67 patients with cerebral adrenoleukodystrophy 14 to 92 months after receipt of gene therapy.
The New...
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were free of major disability. Myelodysplastic syndrome...
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...
