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AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

X-linked Charcot-Marie-Tooth neuropathy (CMTX) is caused by mutations in the gene encoding Gap Junction Protein Beta-1 (GJB1)/Connexin32 (Cx32) in Schwann cells. Neurotrophin-3 (NT-3) is an important autocrine...

Burcak Ozes, Morgan Myers, Kyle Moss, Jennifer Mckinney, Alicia Ridgley… in Gene Therapy (2022)