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 |