Invention Grant
- Patent Title: Treatment of genetic disorders associated with DNA repeat instability
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Application No.: US15855848Application Date: 2017-12-27
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Publication No.: US10689646B2Publication Date: 2020-06-23
- Inventor: Josephus Johannes De Kimpe , Gerard Johannes Platenburg , Derick Gert Wansink
- Applicant: BioMarin Technologies B.V.
- Applicant Address: NL
- Assignee: BioMarin Technologies B.V.
- Current Assignee: BioMarin Technologies B.V.
- Current Assignee Address: NL
- Agency: Lathrop GPM LLP
- Agent James H. Velema, Esq.
- Priority: com.zzzhc.datahub.patent.etl.us.BibliographicData$PriorityClaim@7ddc3ea4 com.zzzhc.datahub.patent.etl.us.BibliographicData$PriorityClaim@3dd58eec
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/11 ; C12Q1/6883 ; A61K48/00

Abstract:
The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
Public/Granted literature
- US20180112214A1 Treatment Of Genetic Disorders Associated With DNA Repeat Instability Public/Granted day:2018-04-26
Information query
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