HTT REPRESSORS AND USES THEREOF

HTT REPRESSORS AND USES THEREOF

Disclosed herein are HTT repressors and methods and compositions for use of these HTT repressors. Disclosed herein are methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. In particular, provided herein are methods and compositions for modifying (e.g., modul...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: CHOI, Vivian, CHIOCCO, Matthew, REBAR, Edward, ZHANG, Lei, FELICE, Brian, MILLER, Jeffrey, PASCHON, David, KLATTE, Debra, ZEITLER, Bryan, FROELICH, Steven, CAREY, Galen, ZHANG, H. Steve
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Beschreibung
Zusammenfassung:Disclosed herein are HTT repressors and methods and compositions for use of these HTT repressors. Disclosed herein are methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. In particular, provided herein are methods and compositions for modifying (e.g., modulating expression of) an HD HTT allele so as to prevent or treat Huntington Disease, including mHTT repressors (that repress mHTT transcripts and thus also repress mHTT protein expression).