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

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...

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Publicado en:Mol Genet Metab
Main Authors: Ikawa, Yasuhiro, Hess, Richard, Dorward, Heidi, Cullinane, Andrew R., Huizing, Marjan, Gochuico, Bernadette R., Gahl, William A., Candotti, Fabio
Formato: Artigo
Idioma:Inglês
Publicado: 2014
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4279856/
https://ncbi.nlm.nih.gov/pubmed/25468649
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.11.006
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