US 11,939,577 B2
Antisense RNA targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease
Liliane Massade, Cachan (FR); Charbel Massaad, Arcueil (FR); Susan Boutary, Arcueil (FR); Giorgia Maria Laura Urbinati, Paris (FR); Patrick Couvreur, Paris (FR); and Didier Desmaële, Fresnes (FR)
Assigned to CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, Paris (FR); and INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris (FR)
Appl. No. 17/279,206
Filed by CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, Paris (FR); and INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), Paris (FR)
PCT Filed Sep. 24, 2019, PCT No. PCT/EP2019/075736
§ 371(c)(1), (2) Date Mar. 24, 2021,
PCT Pub. No. WO2020/064749, PCT Pub. Date Apr. 2, 2020.
Claims priority of application No. 18306241 (EP), filed on Sep. 25, 2018.
Prior Publication US 2022/0002721 A1, Jan. 6, 2022
Int. Cl. C12N 15/113 (2010.01); A61K 9/51 (2006.01); A61K 45/06 (2006.01); A61K 47/69 (2017.01)
CPC C12N 15/113 (2013.01) [A61K 9/5123 (2013.01); A61K 45/06 (2013.01); A61K 47/6929 (2017.08); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2310/351 (2013.01); C12N 2320/32 (2013.01)] 13 Claims
 
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.