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. 2021 Oct 29;11(11):1447. doi: 10.3390/brainsci11111447

Figure 1.

Figure 1

Partial silencing of PMP22 gene (a–d) and gene therapy against CMT1A and CMTX1 (e,f) in different rodent models. (a) ASOs decrease PMP22 mRNA in affected nerves in two CMT1A mouse models. (b) siRNAs conjugated to squalene nanoparticles normalise Pmp22 protein levels in two CMT1A mouse models. (c) AAV serotype 9 viral vectors expressing shRNA directed against PMP22 mRNA restore wild-type expression levels of PMP22 in CMT1A rats. (d) Liposome encapsulated sgRNAs delete the PMP22 TATA-box by CRISPR-Cas9 editing in a mouse model of CMT1A. (e) AAV1.NT-3 gene therapy in transgenic mouse models (TremblerJ and Cx32 knockout mice) and planned in three CMT1A patients. (f) Gene delivery using lentivirus or AAV9 vectors and a myelin-specific MPZ promoter to target murine Schwann cells (Sh3tc2−/− and Cx32 knockout mice). For each approach, the route of administration is also indicated in parentheses.