Abstract: Disclosed herein are compositions and methods for targeting a novel regulatory element of a gene. The compositions may be used in methods of modifying growth of a cell, decreasing cell fitness, increasing cell fitness, and/or treating cancer such as leukemia.

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

Abstract: Disclosed herein are CRISPR/Cas systems comprising a fusion protein and at least one gRNA targeting a gene or a regulatory element thereof in a cell such as an immune cell, and vector compositions encoding the same. The systems and compositions may be used in methods of modulating expression of a gene in a cell such as an immune cell, as well as in methods of treating a disease such as cancer, autoimmune diseases, or viral infections.

Abstract: Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use. The compositions and methods may include gRNAs targeting exons 44 and 55. The compositions and methods may also Include a mutant inverted terminal repeat (ITR) to generate a self-complementary vector genome.

Abstract: Disclosed herein are CRISPR/Cas-based base editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function.

Abstract: Disclosed herein are compositions and methods for inhibiting a gene selected from OGDH, LIPT1, SDHC, and DHRS7B. The inhibitors may be used to activate SNRPN, SPA1, SPA2, or SNORD118, or a combination thereof. The inhibitors may also be used to treat a subject having Prader Willi Syndrome (PWS) or a PWS-like disorder.

Abstract: Disclosed herein are vectors compositions for gene editing of muscle-specific stem cells, or satellite cells, in vivo and methods for treating Duchenne Muscular Dystrophy.

Abstract: Disclosed herein are methods of using probes for high-throughput screening of guide RNA (gRNA) efficiency for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRiSPR-associated (Cas)-based genome editing systems. Further disclosed herein is a humanized transgenic mouse model that recapitulates the severe DMD pathology of human patients. The mouse model may be used for determining the feasibility of CRISPR-based therapies for the correction of the human dystrophin gene by gene editing and methods of use.

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 arm CRISPR/Cas-based genome editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function. The CRISPR/Cas-based genome editing systems may include a guide RNA (gRNA) targeting a fragment of a mutant dystrophin gene, a Cas protein or a fusion protein comprising the Cas protein, and a donor sequence comprising a fragment of a wild-type dystrophin gene.

Abstract: Disclosed herein are systems and methods for the treatment of NF1 in a subject. The CRISPR/Cas-based genome editing systems may include a polynucleotide sequence encoding a guide RNA (gRNA) targeting a fragment of a mutant NF1 gene, a polynucleotide sequence encoding a Cas protein or a fusion protein comprising the Cas protein, and a polynucleotide sequence encoding a donor sequence comprising a fragment of a wild-type NF1 gene.

Abstract: Disclosed herein are fusion proteins for the targeted activation of genes as well as compositions and methods and DMA Targeting Systems comprising the same. The fusion protein may include at least one first polypeptide domain and at least one second polypeptide domain. The first polypeptide domain includes a DMA binding protein, such as a zinc finger protein, a TALE, or a Cas protein, that targets the fusion protein for binding to a specific DNA sequence. The second polypeptide domain includes a modulator of chromatin structure. The fusion protein may further include a third polypeptide domain, the third polypeptide domain including a transcriptional activator domain.

Abstract: 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.

Abstract: The present invention provides DNA targeting systems and methods for delivery of gene editing machinery using lipid nanoparticles or microparticles.

Abstract: Disclosed herein are methods of identifying or engineering a polynucleotide sequence for directing tissue-specific gene expression. The methods may further include creating a regulatory element fragment library. Further disclosed are vectors comprising a tissue-specific regulatory element identified by the methods.

Abstract: Disclosed herein are DNA targeting systems that target a regulatory element of a gene within the 15q11-13 locus. Further provided are DNA targeting systems including at least one gRNA and a Cas9 protein, as well as compositions comprising the same. The compositions may be used in methods for treating Prader-Willi Syndrome (PWS) in a subject. The method may include administering to a subject the DNA targeting system.

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: Disclosed herein are methods and systems for increasing expression of Pax7, methods of activating endogenous myogenic transcription factor Pax7 in a cell, methods of differentiating a stem cell into a skeletal muscle progenitor cell, as well as compositions and methods for treating a subject in need of regenerative muscle progenitor cells. The compositions and methods may include a Cas9-based transcriptional activator protein and at least one guide RNA (gRNA) targeting Pax7.

Abstract: Disclosed herein are compositions, methods, and systems for selecting a polynucleotide for activity as a neuronal-specific transcription factor. The system may include a polynucleotide encoding a reporter protein and a pan-neuronal marker, a Gas protein, and a library of guide RNAs (gRNAs) targeting putative transcription factors. Further provided are methods of screening for a neuronal-specific transcription factor.