Biochemical and physiological improvement in a mouse model of Smith–Lemli–Opitz syndrome (SLOS) following gene transfer with AAV vectors

Smith–Lemli–Opitz syndrome (SLOS) is an inborn error of cholesterol synthesis resulting from a defect in 7-dehydrocholesterol reductase (DHCR7), the enzyme that produces cholesterol from its immediate precursor 7-dehydrocholesterol. Current therapy employing dietary cholesterol is inadequate. As SLO...

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Detalhes bibliográficos
Main Authors: Ying, Lee, Matabosch, Xavier, Serra, Montserrat, Watson, Berna, Shackleton, Cedric, Watson, Gordon
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2014
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4093838/
https://ncbi.nlm.nih.gov/pubmed/25024934
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2014.02.002
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