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Article

In vitro functional correction of Hermansky-Pudlak Syndrome type-1 by lentiviral-mediated gene transfer.

Molecular genetics and metabolism - 1 Jan 2015

Ikawa Yasuhiro, Hess Richard, Dorward Heidi, Cullinane Andrew R, Huizing Marjan, Gochuico Bernadette R, Gahl William A, Candotti Fabio

Abstract excerpt

Hermansky-Pudlak syndrome (HPS) is a genetic disorder characterized by oculocutaneous albinism, bleeding tendency and susceptibility to pulmonary fibrosis. No curative therapy is available. Genetic correction directed to the lungs, bone marrow and/or gastro-intestinal tract might provide alternative forms of treatment for the diseases multi-systemic complications. We demonstrate that lentiviral-mediated gene...

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Source

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Identifiers

PMID: 25468649
DOI: 10.1016/j.ymgme.2014.11.006
PMCID: PMC4279856

Genes

HPS1

Provenance

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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