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1. (WO2018127462) THERAPEUTIC METHOD FOR HUNTINGTON'S DISEASE

Pub. No.:    WO/2018/127462    International Application No.:    PCT/EP2017/084839
Publication Date: Fri Jul 13 01:59:59 CEST 2018 International Filing Date: Sat Dec 30 00:59:59 CET 2017
IPC: C12N 15/113
A61P 25/28
A61K 31/7088
Applicants: ZAIN-LUQMAN, Rula
SMITH, C.I. Edvard
Inventors: ZAIN-LUQMAN, Rula
SMITH, C.I. Edvard
ZAGHLOUL, Eman
MORENO, Pedro
GISSBERG, Olof
LUNDIN, Karin
WENGEL, Jesper
GOOD, Liam
BERGQUIST, Helen
Title: THERAPEUTIC METHOD FOR HUNTINGTON'S DISEASE
Abstract:
The present invention relates to anti-gene oligonucleotides adapted to hybridize to DNA in a HTT gene, which are based on locked nucleic acids, phosphorodiamidate morpholino oligomers, (PMO) or equivalent oligonucleotide analogues comprising a (CAG)n sequence, and whose target is a sequence where the majority of the repeats are CAG/CTG, for use in down regulating the expression of the HTT gene, resulting in reduced HTT mRNA and protein levels in afflicted subjects, or in diagnosis, treatment and/or prevention of Huntington's disease,and where theanti-gene oligonucleotides target non-canonical DNA structures, including hairpin and cruciform. The invention also relates to a delivery system comprising said oligonucleotides and said use thereof.