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Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges

Therapies currently used for hemophilia involve injection of protein concentrates that are expensive, invasive and associated with side effects such as development of neutralizing antibodies (inhibitors) that diminish therapeutic efficacy. Gene transfer is an attractive alternative to circumvent the...

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Autors principals: Raj, Deepak, Davidoff, Andrew M, Nathwani, Amit C
Format: Artigo
Idioma:Inglês
Publicat: 2011
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3200187/
https://ncbi.nlm.nih.gov/pubmed/21939421
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1586/ehm.11.48
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