Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control

Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control

Carbamoyl phosphate synthetase 1 (CPS1) deficiency, a urea-cycle disorder, results in hyperammonemia initiating a sequence of adverse events that can lead to coma and death if not treated rapidly. There is a high unmet need for an effective therapeutic for this disorder, especially in early neonatal...

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Main Authors: Taryn Diep, Wesley Zhou, Rachel E. Reyes, Matthew Nitzahn, Isabel L. Day, Georgios Makris, Lindsay Lueptow, Irina Zhuravka, Stuti Bakshi, Jon Gangoiti, Hyacinth Padaon, Yunfeng Li, Bruce A. Barshop, Johannes Haberle, Gerald S. Lipshutz
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Delivery Strategies
gene therapy
carbamoyl phosphate synthetase deficiency
oversized AAV
ureagenesis
hyperammonemia
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125000241
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