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AssigneeBoard of Regents, The University of Texas System
InventorsViktoriia KYRYCHENKO, Eric N. OLSON, Rhonda BASSEL-DUBY
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CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene and absence or deficiency of dystrophin protein in striated muscle. Provided herein are compositions and methods for treating DMD caused by mutations in the dystrophin Actin Binding Domain 1 (ABD-1). The compositions and method described herein can be used to remove mutant sequences in dystrophin ABD-1 to generate a corrected DMD protein that, while lacking one or more exons (e.g., exons 3-9), retains important functional properties.
A composition comprising:
A genetically engineered mouse whose genome comprises a deletion of exon 8 and 9 of the dystrophin gene resulting in an out of frame shift and a premature stop codon in exon 10.
A genetically engineered mouse produced by a method comprising the steps of:
A method of editing an Actin Binding Domain 1 (ABD-1) dystrophin gene defect in a subject comprising contacting a cell with one or more expression constructs expressing Cas9, a first guide RNA and a second guide RNA, wherein the first guide RNA targets a dystrophin intron 5′ to the gene defect, and the second guide RNA targets a dystrophin intron 3′ to the gene defect, thereby resulting in an edited dystrophin gene lacking dystrophin exons 3-9.
Dependencies are inferred from the claim text.