Perineural delivery of AAV2/9 in non-human primates is a safe and efficient route for gene therapy in Charcot-Marie-Tooth diseases.

ABSTRACT: AAV-based gene therapy represents an attractive treatment for hereditary peripheral neuropathies Charcot-Marie-Tooth diseases. We recently showed that AAV2/9 vector-expressing GFP, locally injected into mouse and rat sciatic nerves, transduced a large amount of myelinating Schwann cells (mSCs). The local delivery of a similar vector expressing a small hairpin RNA (shRNA) targeting PMP22 mRNA prevented CMT1A disease in a rat disease model. Here, we investigated the regional anesthesia standard perineural injection route in non-human primates using AAV2/9 expressing GFP and AAV2/9 expressing shRNA targeting human and monkey PMP22 mRNA. Injecting at multiple sites to cover the largest part of arm and leg nerves, we found that AAV2/9 easily crossed perineurium sheaths to transduce up to 90% of

SUBMITTER: Espallergues J 

PROVIDER: S-EPMC12433476 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature