AAV treatment of Huntington's disease

US11046957B2 · kind B2 · utility

Assignee

• University of Massachusetts · US

Inventors

• Christian Mueller · Wiesbaden, DE
• Neil Aronin · Newton, US
• Edith Pfister · Boxborough, US

Key dates

• Filing date: Sep 17, 2019
• Grant date: Jun 29, 2021
• Expiry date: Sep 17, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14171
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.