Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver



Inherited metabolic diseases (IMDs) of the liver represent a vast and diverse group of rare genetic diseases characterized by the loss or dysfunction of enzymes or proteins essential for metabolic pathways in the liver. Conventional gene therapy involving adeno-associated virus (AAV) serotype 8 vect...

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Detalles Bibliográficos
Autores principales: Bryson, Taylor E., Anglin, Caitlin M., Bridges, P. Hudson, Cottle, Renee N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: YJBM 2017
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5733857/
https://www.ncbi.nlm.nih.gov/pubmed/29259521

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