| abstract |
The present disclosure provides enzyme replacement therapy using gene therapy vectors, such as adeno-associated virus (AAV) vectors expressing human Cystathionine Beta-Synthase (CBS) to reduce the amount of serum homocysteine (Hcy) and increase the amount of downstream metabolites, such as cystathionine and cysteine (Cys), which can be used for treatment of diseases, such as homocystinuria and homocysteine remethylation disorders. |