Invention Grant
- Patent Title: Therapeutic method for huntington's disease
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Application No.: US16475642Application Date: 2017-12-29
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Publication No.: US11268093B2Publication Date: 2022-03-08
- Inventor: Rula Zain-Luqman , C. I. Edvard Smith , Eman Zaghloul , Pedro Moreno , Olof Gissberg , Karin Lundin , Jesper Wengel , Liam Good , Helen Bergquist
- Applicant: Rula Zain-Luqman , C. I. Edvard Smith
- Applicant Address: SE Taby; SE Stockholm
- Assignee: Rula Zain-Luqman,C. I. Edvard Smith
- Current Assignee: Rula Zain-Luqman,C. I. Edvard Smith
- Current Assignee Address: SE Taby; SE Stockholm
- Agency: Kim IP Law Group LLC
- Priority: SE1750001-8 20170103
- International Application: PCT/EP2017/084839 WO 20171229
- International Announcement: WO2018/127462 WO 20180712
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C12N15/113 ; A61P25/28 ; A61K31/7088 ; A61K47/42 ; C12Q1/6883

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 the anti-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.
Public/Granted literature
- US20210024922A1 THERAPEUTIC METHOD FOR HUNTINGTON'S DISEASE Public/Granted day:2021-01-28
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