New Review Highlights Advances and Ongoing Challenges in Gene Therapy for Neurologic Disorders
June 2, 2025
A new review article, “Current Advances and Challenges in Gene Therapies for Neurologic Disorders,” was recently published in Neurology: Genetics. The lead author of this comprehensive primer was UPMC Children’s Hospital of Pittsburgh neurologist Deepa S. Rajan, MD.
The review provides clinicians with an overview of the current state of gene-targeted treatments for rare neurologic diseases, including the mechanisms, regulatory approvals, and post-treatment monitoring associated with approved therapies for conditions such as spinal muscular atrophy, metachromatic leukodystrophy, and adrenoleukodystrophy. The paper also explores delivery challenges, safety considerations, and emerging approaches that may shape the future of care.
Dr. Rajan is director of the UPMC Children’s Center for NeuroGenomics and director of the Program for the Study of Neurodevelopment in Rare Disorders. She is an associate professor of Pediatrics at the University of Pittsburgh School of Medicine and her clinical and research work focuses on rare neurogenetic disorders and the integration of novel therapies into clinical practice.
A Q&A with Dr. Rajan about the review and its clinical implications was recently published on Physician’s Weekly.
Reference
Porcari GS, Collyer JW, Adang LA, Rajan DS. Current Advances and Challenges in Gene Therapies for Neurologic Disorders. Neurol Genet. 2025; 11: e200229
