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Article

Intrathecal gene therapy rescues a model of demyelinating peripheral neuropathy

28 Mar 2016

Abstract excerpt

Inherited demyelinating peripheral neuropathies are progressive incurable diseases without effective treatment. To develop a gene therapy approach targeting myelinating Schwann cells that can be translatable, we delivered a lentiviral vector using a single lumbar intrathecal injection and a myelin-specific promoter. The human gene of interest, GJB1, which is mutated in X-linked Charcot-Marie-Tooth Disease...

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PMID: 27035961
DOI: 10.1073/pnas.1522202113

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