Abstract: 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.

Abstract: Disclosed herein are methods of using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based epigenomic editing systems for high-throughput screening of regulatory element function.

Abstract: 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.

Abstract: Disclosed herein are compositions and methods for programming immune cell function though targeted gene regulation.

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

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

Abstract: Disclosed herein are vectors that targets a dystrophin gene, encoding at least one Cas9 molecule or a Cas9 fusion protein, and at least one gRNA molecule (e.g., two gRNA molecules), and compositions and cells comprising such vectors. Also provided are methods for using the vectors, compositions and cells for genome engineering (e.g., correcting a mutant dystrophin gene), and for treating DMD.

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

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

Abstract: 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.

Abstract: 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.

Abstract: Disclosed herein are methods of using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based epigenomic editing systems for high-throughput screening of regulatory element function.

Abstract: 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.

Abstract: 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.

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

Abstract: 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.

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

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

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

Abstract: 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.