Duke University

University of Utah Research Foundation

Jonathan M Brunger

Charles A Gersbach

Farshid Guilak

Niloofar Farhang

Robert Daniel Bowles

Joshua Stover

Lori Setton

Disclosed herein are compositions and methods for cell therapy comprising an engineered cell. The present invention is directed to a composition for treating a subject having or suspected of having a disease, the composition comprising a modified cell comprising a modified endogenous gene, wherein an endogenous gene or fragment thereof is replaced with a transgene using a CRISPR/Cas9 system to generate the modified endogenous gene, the modified cell having an altered response to a cell signal or stimulus.

Compositions, systems and methods for cell therapy

Described herein are compositions and methods for treatment and prevention of low back pain. The compositions include vectors comprising nucleotide sequences encoding one or more CRISPR-Cas system guide RNAs and a RNA-directed nuclease. The methods include modulating expression of a gene in a cell using said compositions, introducing a CRISPR-Cas system into a cell comprising one or more vectors comprising said compositions, inducing site-specific DNA cleavage in a cell, and treating a subject having lower back pain, and lower back pain caused by degenerative disc disease using the compositions disclosed herein.

RNA-guided transcriptional regulation and methods of using the same for the treatment of back pain

Growing community of inventors

Durham, NC, United States of America

Jonathan M Brunger