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: 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 one or more conditions associated with ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 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 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 application provides materials and methods for treating a patient with one or more conditions or disorders associated with AGXT, both ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Primary Hyperoxaluria Type 1 (PH1). The present application also provides materials and methods for editing an AGXT gene in a cell by genome editing. The present application also provides materials and methods for altering a contiguous genomic sequence of an AGXT gene in a cell. In addition, the present application provides one or more gRNAs for editing an AGXT gene. The present application also provides a therapeutic comprising at least one or more gRNAs for editing an AGXT gene. In addition, the present application provides a therapeutic for treating a patient with an AGXT 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: 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: 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 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: The present disclosure provides materials and methods for treating a patient with one or more conditions associated with SOD1 whether ex vivo or in vivo. In addition, the present disclosure provides materials and methods for editing and/or modulating the expression of SOD1 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: 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: 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.

Abstract: The present application provides materials and methods for treating a patient with one or more condition associated with FXN whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of FXN 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 SCN9A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN9A 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: Provided include materials and methods for treating a subject with one or more conditions associated with WAS gene whether ex vivo or in vivo. Also provided include materials and methods for editing and/or modulating the expression of WAS gene in a cell by genome editing.