Correction of Ornithine Transcarbamylase (OTC) Deficiency in Spf-Ash Mice by Introduction of Rat OTC Gene

    February 1991 in “ FEBS Letters ”
    Tokihiko Shimada, Takashi Noda, Masaaki Tashiro, Takashi Murakami, Masaki Takiguchi, Masataka Mori, Ken–ichi Yamamura, Takeyori Saheki
    The study introduced the rat ornithine transcarbamylase (OTC) gene into OTC-deficient spf-ash mice, resulting in significant improvements in enzyme activity and metabolic markers. Hemizygous spf-ash mice with the transgene exhibited liver OTC activity about twice that of nontransgenic counterparts and small intestinal OTC activity 6 times higher, reaching 12% and 27% of control levels, respectively. These transgenic mice displayed normal hair growth, nearly normalized urinary orotic acid excretion, and normalized serum citrulline concentration, indicating a successful correction of the OTC deficiency.
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