Methods for the treatment of Danon disease and other disorders of autophagy

US11065347B2 · kind B2 · utility

Assignee

• The Regents of the University of California · US

Inventors

• Eric Adler · Jericho, US
• Bradley Nelson · St. George, US
• Sherin Hashem · Village of La Jolla, US

Key dates

• Filing date: Jan 19, 2017
• Grant date: Jul 20, 2021
• Expiry date: Jan 19, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

This disclosure provides gene therapy vectors comprising a polynucleotide encoding one or more isoforms of lysosome-associated membrane protein 2 (LAMP-2), and methods of using such gene therapy vectors for the treatment of Danon disease and other autophagy disorders.