METHODS AND COMPOSITIONS OF OTC CONSTRUCTS AND VECTORS

摘要

Provided herein are methods and compositions related to nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising an OTC codon-optimized sequence, as well as related vectors, such as AAV vectors. Also, provided are methods for administering AAV vectors that comprise a sequence that encodes an enzyme associated with an urea cycle disorder and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant.