Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

Abstract: Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.

Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.

Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.

Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.

Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.

Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

Abstract: The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN2 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Spinocerebellar ataxia type 1 (SCA2). Also provided are materials and methods for editing a ATXN2 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a ATXN2 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a ATXN2 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a ATXN2 gene. In addition, the present disclosure provides therapeutics for treating patients with a ATXN2 related condition or disorder.

Abstract: The present application provides materials and methods for treating a patient with autosomal dominant RP, both ex vivo and in vivo; materials and methods for editing a RHO gene in a human cell; and materials and methods for editing a P23H mutation in a RHO gene in a human cell. In addition, the present application provides one or more gRNAs or sgRNAs for editing a RHO gene; one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene. The present application provides a therapeutic for treating a patient with autosomal dominant RP. The present application provides a kit for treating a patient with autosomal dominant RP. In addition, the present application provides a self-inactivating CRISPR-Cas system.

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with APOCIII whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of APOCIII gene in a cell by genome editing.

Abstract: The present application provides materials and methods for treating a patient with one or more conditions or disorders associated with LAMA2, both ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Merosin-deficient Cogenital Muscular Dystrophy (MDCMD). The present application also provides materials and methods for editing a LAMA2 gene in a cell by genome editing. The present application also provides materials and methods for altering a contiguous genomic sequence of a LAMA2 gene in a cell. In addition, the present application provides one or more gRNAs for editing a LAMA2 gene. The present application also provides a therapeutic comprising at least one or more gRNAs for editing a LAMA2 gene. In addition, the present application provides a therapeutic for treating a patient with a LAMA2 related condition or disorder.

Abstract: The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with SNCA whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Early Onset Parkinson's Disease (PARK1). Also provided are materials and methods for editing a SNCA gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a SNCA gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a SNCA gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a SNCA gene. In addition, the present disclosure provides therapeutics for treating patients with a SNCA related condition or disorder.

Abstract: The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with COL7A1 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Dystrophic Epidermolysis Bullosa (DEB). Also provided are materials and methods for editing a COL7A1 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a COL7A1 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a COL7A1 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a COL7A1 gene. In addition, the present disclosure provides therapeutics for treating patients with a COL7A1 related condition or disorder.

Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for creating permanent changes to the genome that can result in at least one deletion, insertion, modulation, or inactivation of a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

Abstract: Materials and methods for treating a patient with hemoglobinopathy, both ex vivo and in vivo and materials and methods for deleting at least a portion of a human beta globin locus on chromosome 11 in a human cell by genome editing and thereby increasing the production of fetal hemoglobin (HbF).

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with SCN10A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN10A gene in a cell by genome editing.

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.

Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL4 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL4 gene in a cell by genome editing.