Abstract: Methods and systems for searching genomes for potential nucleotide-guided nuclease off-target sites are provided. Also provided are methods of searching genomes for potential off-target deadCas9 binding sites. In some embodiments, the methods include ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the DNA, RNA, or DNA/RNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome, allowing the selection of better target sites and/or experimental confirmation of off-target sites.

Abstract: The disclosure provides to CRISPR/Cas systems and compositions which target the dystrophin gene. Also provided are methods for using the CRISPR/Cas systems, vectors and compositions in methods for genome engineering to correct a mutant dystrophin gene, and for treating Duchenne muscular dystrophy.

Abstract: The invention relates to self-inactivating/self-targeting CRISPR/Cas or CRISPR/Cpf1 systems. The invention also relates to genetically modified cells related to the same. The invention also relates to methods of controlling Cas9 expression in a cell related to the same. The invention also relates to methods of genetically modifying a cell related to the same. The invention also relates to nucleic acids for use in a self-inactivating CRISPR/Cas or CRISPR/Cpf1 system. The invention also relates to pharmaceutical compositions related to the same.

Abstract: Methods and systems for searching genomes for potential nucleotide-guided nuclease off-target sites are provided. Also provided are methods of searching genomes for potential off-target deadCas9 binding sites. In some embodiments, the methods include ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the DNA, RNA, or DNA/RNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome, allowing the selection of better target sites and/or experimental confirmation of off-target sites.

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and /or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and/or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.

Abstract: The present application provides materials and methods for treating a patient with severe combined immunodeficiency (SCID) or Omenn Syndrome, both ex vive and in vive. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the Recombination Activating Gene 1 (RAG1) gene in a cell by genome editing.

Abstract: The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and ?-thalassemia.

Abstract: Methods and systems for searching genomes for potential CRISPR off-target sites are provided. In preferred embodiments, the methods include identifying possible on- and off-target cleavage sites and/or ranking the potential off-target sites based on the number and location of mismatches, insertions, and/or deletions in the gRNA guide sequence relative to the genomic DNA sequence at a putative target site in the genome. These methods allow for the selection of better target sites and/or experimental confirmation of off-target sites and are an improvement over partial search mechanisms that fail to locate every possible target site.