Patent ID: 11939577
Assignee: CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method for treating Charcot-Marie-Tooth 1A (CMT-1A), wherein said method comprises administering to a subject in need thereof an antisense RNA targeting an mRNA encoding PMP22 protein, wherein said antisense RNA is a siRNA, wherein said antisense RNA has a length of from 12 to 50 nucleotides and wherein said antisense RNA is complementary to a portion consisting of or comprised within:
nucleotides 989 to 1007 of sequence SEQ ID NO: 11,
nucleotides 970 to 988 of sequence SEQ ID NO: 9,
nucleotides 1721 to 1739 of sequence SEQ ID NO: 11,
nucleotides 1726 to 1744 sequence SEQ ID NO: 9,
nucleotides 431 to 449 of sequence SEQ ID NO: 11,
nucleotides 429 to 447 sequence SEQ ID NO: 9,
nucleotides 1805 to 1823 of sequence SEQ ID NO: 11,
nucleotides 1809 to 1827 sequence SEQ ID NO: 9,
nucleotides 921 to 939 of sequence SEQ ID NO: 11, or
nucleotides 903 to 921 sequence SEQ ID NO: 9.