Methods and compositions of OTC constructs and vectors

摘要

Provided herein are nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising OTC codon-optimized sequences, as well as methods and compositions relating to related vectors such as AAV vectors. Also provided is a method of administering an AAV vector comprising a sequence encoding an enzyme associated with urea cycle disorder and an expression control sequence in combination with a synthetic nanocarrier coupled to an immunosuppressant.