P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin

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Bibliographic Details
Main Authors: Jerry Vockley, Shakuntala Basu, Xue-Jun Zhao, Bianca Seminotti, Clinton Van't Land, Al-Walid Mohsen
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Genetics in Medicine Open
Online Access:http://www.sciencedirect.com/science/article/pii/S2949774425009021
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