Enantiomeric compound for the reduction of the deleterious activity of extended nucleotide repeat containing genes

US10882821B1 · kind B1 · utility

Assignee

• The Board of Trustees of the Leland Stanford Junior University · US

Inventors

• Stanley N. Cohen · Stanford, US
• Ning Deng · Cambridge, US

Key dates

• Filing date: Sep 20, 2018
• Grant date: Jan 5, 2021
• Expiry date: Sep 28, 2038

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61P25/28
• WIPO field: Organic fine chemistry
• WIPO sector: Chemistry

Abstract

Aspects of the present disclosure include methods of reducing the deleterious impact of a target gene in a cell, such as 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 an enantiomeric tetrahydrocarbazolamine compound. 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, 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.