Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes

US10675293B2 · kind B2 · utility

Assignees

• The Board of Trustees of the Leland Stanford Junior University · US
• NATIONAL YANG-MING UNIVERSITY · TW

Inventors

• Stanley N. Cohen · Stanford, US
• Ning Deng · Cambridge, US
• Yanan Feng · San Jose, US
• Tzu-Hao Cheng · Taipei, TW
• Yun Wu · Taipei, TW
• Wen-Chieh Hsieh · Tainan, TW

Key dates

• Filing date: May 18, 2016
• Grant date: Jun 9, 2020
• Expiry date: May 18, 2036

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K48/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Aspects of the invention include methods of reducing the deleterious activity of a mutant extended nucleotide repeat (NR) containing target gene in a cell by contacting the cell with an effective amount of a nucleoside agent, as well as compositions used in such methods. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended NR containing target gene may be reduced in a variety of different ways, e.g., by reducing (and in some instances differentially, including selectively, reducing) the production or activity of toxic expression products (e.g., RNA or protein) encoded by the target gene. Kits and compositions for practicing the subject methods are also provided.