Article

A dose-escalation and safety study of gene therapy for CMT4C neuropathy

2026-01-13

Abstract excerpt

<title>Abstract</title> <p> Charcot-Marie-Tooth disease type 4C is a demyelinating neuropathy caused by loss of function mutations in the <italic>SH3TC2</italic> gene, that is highly expressed in myelinating Schwann cells. We generated and tested a clinical stage vector with a minimal human MPZ promoter driving expression of <italic>SH3TC2</italic> . Groups of 1-month old <italic>Sh3tc2</italic> <sup>−/−</...

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Literature Corpus work: 06a5bb16-2c07-561d-8b95-cf4a507bcb01
DOI: 10.21203/rs.3.rs-8334328/v1

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