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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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| Udgivet i: | J Inherit Metab Dis |
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| Main Authors: | , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2019
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| Fag: | |
| Online adgang: | 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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