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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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Библиографические подробности
Главные авторы: Raj, Deepak, Davidoff, Andrew M, Nathwani, Amit C
Формат: Artigo
Язык:Inglês
Опубликовано: 2011
Предметы:
Online-ссылка: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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