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W19-005: Gene Editing Strategies for Treatment of Feline Hypertrophic Cardiomyopathy

Feline hypertrophic cardiomyopathy (HCM)is a common disease of cats that results from heart muscle tissue thickening due to changes arising from genetic mutations in certain breeds. This thickening leads to improper blood flow, which may cause untreatable heart disease leading to mortality. While this disease is found in 10-15% of cats, HCM is more common in certain breeds like Maine Coon cats. One of these deleterious traits includes a mutation in a particular protein, which helps with the contractility of the heart, that is the result of a single gene mutation in Maine Coon cats. This study proposes to use a new genetic therapy, CRISPR-Cas9, to replace a mutated gene with a normal gene. There are many potential benefits to using CRISPR-Cas9 to target HCM in Maine Coon cats. First and foremost, CRISPR technologies are very specific so there is unlikely to be non-specific targets. Second, HCM in Maine Coon cats is caused by a single point mutation, so there is a reduced chance to impact other parts of the genome. This study is designed to test this technique on cultured cells as a first step to potentially eradicating this devastating disease from the Maine Coon cat breed.

Grant ID: W19-005

Status: Active

Year Funded: 2019

Amount awarded: $15,000

Investigator: Dr. Elliott Chiu, Colorado State University