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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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Detalhes bibliográficos
Main Authors: Raj, Deepak, Davidoff, Andrew M, Nathwani, Amit C
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
Assuntos:
Acesso em linha: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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