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AAV-based Neonatal Gene Therapy for Hemophilia A: Long-Term Correction and Avoidance of Immune Responses in Mice
Hemophilia A gene therapy has been hampered by immune responses to vector-associated antigens and by neutralizing antibodies or inhibitors to the factor VIII (FVIII) protein; these ‘inhibitors’ more commonly effect hemophilia A patients than those with hemophilia B. A gene replacement strategy begin...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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2012
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3432168/ https://www.ncbi.nlm.nih.gov/pubmed/22241178 http://dx.doi.org/10.1038/gt.2011.200 |