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Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome

Liver metabolism disorders are attractive targets for gene therapy, because low vector doses can reverse the buildup of toxic metabolites in the blood. Crigler-Najjar syndrome is an inherited disorder of bilirubin metabolism that is caused by the absence of uridine diphosphate glucuronosyl transfera...

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Detalles Bibliográficos
Autores principales:	Greig, Jenny A., Nordin, Jayme M.L., Draper, Christine, McMenamin, Deirdre, Chroscinski, Edward A., Bell, Peter, Gray, John T., Richman, Laura K., Wilson, James M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6090885/ https://www.ncbi.nlm.nih.gov/pubmed/30112420 http://dx.doi.org/10.1016/j.omtm.2018.07.008

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