Pharmaceutical composition for treating Charcot Marie Tooth disease
US10119142B2 · kind B2 · utility
2Cited by
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12Claims
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Key dates
| Filing date | Aug 30, 2017 |
| Grant date | Nov 6, 2018 |
| Priority date | — |
| Expiry date | Aug 30, 2037 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/34
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
An siRNA for specifically targeting a PMP22 mutant gene and a pharmaceutical composition for preventing or treating Charcot Marie Tooth disease, which includes the same, are provided. According to the present invention, it is confirmed that selective suppression of a PMP22 mutant allele by a non-viral delivery system of siRNA may improve demyelinating neuropathic symptoms of CMT in vivo, enhance a motor ability and increase a volume of muscle. Therefore, the siRNA may be used in a useful method for treating various dominantly inherited peripheral neuropathies including CMT.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.