MATERIALS AND METHODS FOR TREATMENT OF MEROSIN-DEFICIENT COGENITAL MUSCULAR DYSTROPHY (MDCMD) AND OTHER LAMININ, ALPHA 2 (LAMA2) GENE RELATED CONDITIONS OR DISORDERS

摘要

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.