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AAV9 Gene Replacement Therapy for Respiratory Insufficiency in Very-long Chain Acyl-CoA Dehydrogenase Deficiency

Very-long chain acyl-CoA dehydrogenase (VLCAD) deficiency (VLCADD) is an autosomal recessive disorder of fatty acid oxidation. Fatty acids are a major source of energy during catabolic stress, so the abscense of VLCAD can result in a metabolic crises and respiratory insufficiency. The etiology of th...

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Bibliografski detalji
Izdano u:J Inherit Metab Dis
Glavni autori: Zieger, Marina, Keeler, Allison M., Flotte, Terence R., ElMallah, Mai K.
Format: Artigo
Jezik:Inglês
Izdano: 2019
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6739149/
https://ncbi.nlm.nih.gov/pubmed/30993714
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12101
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