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Muscle-directed Gene Therapy for Hemophilia B with More Efficient and Less Immunogenic AAV Vectors
BACKGROUND: Adeno-associated viral vector (AAV)-mediated and muscle-directed gene therapy is a safe and noninvasive approach to treat hemophilia B and other genetic diseases. However, low efficiency of transduction, inhibitor formation and high prevalence of pre-existing immunity to the AAV capsid i...
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| Main Authors: | , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2011
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3393098/ https://ncbi.nlm.nih.gov/pubmed/21883883 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1538-7836.2011.04491.x |
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