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Article

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

The Yale journal of biology and medicine - 1 Dec 2017

Bryson Taylor E, Anglin Caitlin M, Bridges P Hudson, Cottle Renee N

Abstract excerpt

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 vectors provide therapeutically high levels of hepatic transgene expression facilitating the correction of the disease...

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Identifiers

PMID: 29259521
PMCID: PMC5733857

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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