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...

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Detalles Bibliográficos
Autores principales: Hu, Chuhong, Lipshutz, Gerald S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2012
Materias:
Article
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

Internet

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

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