Duke University

North Carolina State University

University of Utah Research Foundation

Editas Medicine, Inc.

Charles A Gersbach

David A Bumcrot

Isaac B Hilton

David G Ousterout

Pablo Perez-Pinera

Pratiksha I Thakore

Farshid Guilak

Joshua B Black

Chase Beisel

Ami M Kabadi

Jonathan M Brunger

Adrian Pickar Oliver

Niloofar Farhang

Robert Daniel Bowles

Timothy E Reddy

Sarina Madhavan

Jacqueline N Robinson-Hamm

Christopher Nelson

Piotr Marszalek

Dewran Kocak

Eric Josephs

Tyler S Klann

Gregory E Crawford

Lori Setton

Joshua Stover

Nicholas C Huston

The present disclosure provides Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use.

CRISPR/Cas9-based repressors for silencing gene targets in vivo and methods of use

Disclosed herein are CRISPR/Cas9-based gene activation systems that include a fusion protein of a Cas9 protein and a protein having histone acetyltransferase activity, and methods of using said systems.

Compositions and methods for epigenome editing

Disclosed herein are compositions of transcription activator-like effectors transcription factors and methods of using the compositions for inducing gene expression of mammalian genes.

Compositions and methods for the induction and tuning of gene expression

Disclosed herein are therapeutic applications of CRISPR/Cpf1-based genome editing.

Therapeutic applications of CPF1-based genome editing

Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.

Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

Disclosed herein are transcription activator-like effector nuclease (TALEN)-related compositions and methods of using said TALENs for correcting mutant genes.

Genetic correction of mutated genes

Disclosed herein are Type I Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) system related compositions and methods of using said Type I CRISPR/Cas system related compositions for altering gene expression and genome engineering. The invention relates to compositions comprising Type I CRISPR-Cas polypeptides and CRISPR array nucleic acids designed for genome modification in eukaryotic cells and for targeted killing of eukaryotic cells.

Genome engineering with Type I CRISPR systems in eukaryotic cells

Disclosed herein are optimized guide RNAs (gRNAs) that have increased target binding specificity and reduced off-target binding. Further disclosed herein are methods of designing and using the optimized gRNAs.

Compositions and methods of improving specificity in genomic engineering using RNA-guided endonucleases

Genome engineering with type I CRISPR systems in eukaryotic cells

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

Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.

Growing community of inventors

Durham, NC, United States of America

Charles A Gersbach