AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C.

Comparison of NF-L levels in early-treated mice (n = 4 per group) showed a trend for improvement in treated compared with mock-treated 3-month-old Sh3tc2 −/− mice but did not reach WT levels ( Figure S4 B), and a similar trend for NF-L reduction in late-treated 8-month-old animals compared with the corresponding mock group approaching WT levels ( Figure S4 C).

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