Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control
Mol Ther Nucleic Acids. 2025 Feb 3;36(1):102470. doi: 10.1016/j.omtn.2025.102470. eCollection 2025 Mar 11. ABSTRACT Carbamoyl phosphate synthetase 1 (CPS1) deficiency, a urea-cycle disorder, results in …