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In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.

Human gene therapy - 1 Aug 2018

Song Chun-Qing, Wang Dan, Jiang Tingting, O'Connor Kevin, Tang Qiushi, Cai Lingling, Li Xiangrui, Weng Zhiping, Yin Hao, Gao Guangping, Mueller Christian, Flotte Terence R, Xue Wen

Abstract excerpt

CRISPR (clustered regularly interspaced short palindromic repeats) genome editing holds promise in the treatment of genetic diseases that currently lack effective long-term therapies. Patients with alpha-1 antitrypsin (AAT) deficiency develop progressive lung disease due to the loss of AAT's antiprotease function and liver disease due to a toxic gain of function of the common mutant allele. However, it remains...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 29597895
DOI: 10.1089/hum.2017.225
PMCID: PMC6110121

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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