| abstract |
Provided herein are methods and compositions related to nucleic acids encoding methylmalonyl-CoA mutase (MUT) as well as related vectors, such as AAV vectors and Anc80 vectors. Also, provided are methods for administering viral vectors that comprise a sequence that encodes an enzyme associated with an organic acidemia and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant. |