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Article

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

28 Oct 2013

Abstract excerpt

Charcot-Marie-Tooth (CMT) neuropathies represent a heterogeneous group of peripheral nerve disorders affecting 1 in 2,500 persons. One variant, CMT1A, is a primary Schwann cell (SC) disorder, and represents the single most common variant. In previous studies, we showed that neurotrophin-3 (NT-3) improved the trembler(J) (Tr(J)) mouse and also showed efficacy in CMT1A patients. Long-term treatment with NT-3 was...

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PMID: 24162799
DOI: 10.1038/mt.2013.250

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