TLDR Introducing the rat OTC gene normalized hair growth in SPF-ASH mice.
The study focused on the sparse fur-abnormal skin and hair (SPF-ASH) mouse, a model for human ornithine transcarbamylase (OTC) deficiency, which exhibited symptoms like abnormal skin, delayed hair growth, and metabolic issues. Researchers introduced the rat OTC gene into SPF-ASH mice, resulting in hemizygous mice with normalized hair growth, normal metabolic markers, and increased OTC activity in the liver and small intestine compared to SPF-ASH mice. This demonstrated the potential of gene therapy in correcting the symptoms associated with OTC deficiency in this mouse model.
14 citations,
February 1991 in “FEBS Letters” Introducing the rat OTC gene partially corrected OTC deficiency in mice.
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