Methods and products for increasing CFTR

US10934561B1 · kind B1 · utility

Assignee

• Board of Trustees of the Univeristy of Alabama, for and on behalf of the University of Alabama in Huntsville · US

Inventors

• Eric Mendenhall · Rancho Santa Margarita, US
• Candice Joyce Coppola · Huntsville, US

Key dates

• Filing date: Oct 27, 2017
• Grant date: Mar 2, 2021
• Expiry date: Dec 9, 2037

Classification

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

Abstract

Cystic Fibrosis (CF) is a recessive, lethal genetic disease in which many patients have inherited DNA mutations that create a CFTR protein that is produced, but ineffective in its function. The present disclosure generally pertains to methods and products for increasing CFTR production through the use of a CRISPR/dCas9 system. This disclosure describes such system, which comprises gRNA configured to target the CFTR domain and dCas9 configured to upregulate expression of the gRNA target, and methods of using the same.