Pharmaceutical composition for treating Charcot Marie Tooth disease
AssigneeSamsung Life Public Welfare Foundation
InventorsByung-Ok Choi, Young Bin Hong
Life of this patent
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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.
Claims12
A method of treating Charcot Marie Tooth disease in a subject in need thereof, comprising:
- administering to the subject an effective amount of a pharmaceutical composition comprising an expression inhibitor of a peripheral myelin protein 22 (PMP22) mutant gene as an active ingredient,
- wherein the expression inhibitor is a small interfering RNA (siRNA) haying a sense strand selected from the group consisting of SEQ ID NOs:
- 2, 3, and 4, and an antisense strand consisting of a sequence complementary to the sense strand.
An siRNA having a sense strand selected from the group consisting of SEQ ID NOs:
- 2, 3, and 4 and an antisense strand consisting of a sequence complementary to the sense strand and binding to a PMP22 mutant gene to suppress expression of the PMP22 mutant gene.
Dependencies are inferred from the claim text.