Gene therapies validated in animal models of IPN

Type Target Therapeutic gene Mode of action Vector Route Reference
Gene addition CMT1A Neurotrophin-3 Stimulation of neurite outgrowth and myelination AAV Intramuscular 50
Gene replacement CMT4C SH3TC2 Replacement of the autosomal recessive gene Lentivirus Intrathecal 36
CMTX1 GJB1 Delivery of a wild-type gene Lentivirus Intraneural (sciatic nerve) 41
Delivery of a wild-type gene Lentivirus Intrathecal 42, 43
Gene silencing CMT1E siRNA Targeting and suppressing the mutant allele of PMP22 Naked (synthetic siRNA) Intraperitoneal 61
CMT1A miRNA (miR-381) Downregulating PMP22 overexpression Lentivirus Intraneural (sciatic nerve) 69
Antisense oligonucleotide Downregulating PMP22 overexpression by exon skipping Naked (synthetic oligonucleotide) Subcutaneous 80
Gene editing CMT1A CRISPR/Cas9 Downregulating PMP22 overexpression by disrupting TATA-box Naked (protein/gRNA) Intraneural (sciatic nerve) 82
Exp Neurobiol 2020;29:177~188
© Exp Neurobiol