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Sustained Expression of α(1)-Antitrypsin after Transplantation of Manipulated Hematopoietic Stem Cells
Inherited mutations in the human α(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...
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| Main Authors: | , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Thoracic Society
2008
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2542452/ https://ncbi.nlm.nih.gov/pubmed/18323534 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1165/rcmb.2007-0133OC |
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