Adeno-associated virus gene therapy to the rescue for Charcot-Marie-Tooth disease type 4J
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://www.jci.org/articles/view/149492/files/pdf
Reference20 articles.
1. Genetics of Charcot-Marie-Tooth (CMT) Disease within the Frame of the Human Genome Project Success
2. Next-generation sequencing in Charcot–Marie–Tooth disease: opportunities and challenges
3. Peripheral neuropathy in complex inherited diseases: an approach to diagnosis
4. Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy
5. Gene-Targeting Therapeutics for Neurological Disease: Lessons Learned from Spinal Muscular Atrophy
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