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Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively. Recently, gene therapy clinical trials with adeno-associated virus (AAV) vectors and protein-engineered transgenes, B-do...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2018
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6349562/ https://www.ncbi.nlm.nih.gov/pubmed/30705923 http://dx.doi.org/10.1016/j.omtm.2018.12.007 |