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piggyBac-mediated phenotypic correction of factor VIII deficiency
Hemophilia A, caused by a deficiency in factor VIII (FVIII), is the most severe inherited bleeding disorder. Hemophilia A is an attractive gene therapy candidate because even small increases in FVIII levels will positively alter the phenotype. While several vectors are under investigation, gene addi...
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| Publicado no: | Mol Ther Methods Clin Dev |
|---|---|
| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4362371/ https://ncbi.nlm.nih.gov/pubmed/26015980 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.42 |
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