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Article

Sustained expression of alpha1-antitrypsin after transplantation of manipulated hematopoietic stem cells.

American journal of respiratory cell and molecular biology - 1 Aug 2008

Wilson Andrew A, Kwok Letty W, Hovav Avi-Hai, Ohle Sarah J, Little Frederic F, Fine Alan, Kotton Darrell N

Abstract excerpt

Inherited mutations in the human alpha(1)-antitrypsin (AAT) gene lead to deficient circulating levels of AAT protein and a predisposition to developing emphysema. Gene therapy for individuals deficient in AAT is an attractive goal, because transfer of a normal AAT gene into any cell type able to secrete AAT should reverse deficient AAT levels and attenuate progression of lung disease. Here we present an approach...

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Identifiers

PMID: 18323534
DOI: 10.1165/rcmb.2007-0133OC
PMCID: PMC2542452

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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