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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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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author Jerry Vockley
Shakuntala Basu
Xue-Jun Zhao
Bianca Seminotti
Clinton Van't Land
Al-Walid Mohsen
author_facet Jerry Vockley
Shakuntala Basu
Xue-Jun Zhao
Bianca Seminotti
Clinton Van't Land
Al-Walid Mohsen
author_sort Jerry Vockley
collection DOAJ
format Article
id doaj-art-61d6844f7ef44b41aef574d0070768a2
institution DOAJ
issn 2949-7744
language English
publishDate 2025-01-01
publisher Elsevier
record_format Article
series Genetics in Medicine Open
spelling doaj-art-61d6844f7ef44b41aef574d0070768a22025-08-20T03:14:00ZengElsevierGenetics in Medicine Open2949-77442025-01-01310286310.1016/j.gimo.2025.102863P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoinJerry Vockley0Shakuntala Basu1Xue-Jun Zhao2Bianca Seminotti3Clinton Van't Land4Al-Walid Mohsen5UPMC Children's Hospital of PittsburghUniversity of Pittsburgh School of MedicineUniversity of Pittsburgh School of MedicineUniversity of Pittsburgh School of MedicineUniversity of Pittsburgh School of MedicineUniversity of Pittsburgh School of Medicinehttp://www.sciencedirect.com/science/article/pii/S2949774425009021
spellingShingle Jerry Vockley
Shakuntala Basu
Xue-Jun Zhao
Bianca Seminotti
Clinton Van't Land
Al-Walid Mohsen
P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
Genetics in Medicine Open
title P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
title_full P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
title_fullStr P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
title_full_unstemmed P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
title_short P019: Comparison of treatment efficacy of very long chain acyl-CoA (VLCAD) deficiency with an AAV9.hVLCAD vector, synthetic VLCAD mRNA, and triheptanoin
title_sort p019 comparison of treatment efficacy of very long chain acyl coa vlcad deficiency with an aav9 hvlcad vector synthetic vlcad mrna and triheptanoin
url http://www.sciencedirect.com/science/article/pii/S2949774425009021
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