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Publication Detail
Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Journal Article
  • Authors:
    Raj D, Davidoff AM, Nathwani AC
  • Publication date:
  • Pagination:
    539, 549
  • Journal:
    Expert Rev Hematol
  • Volume:
  • Issue:
  • Status:
  • Country:
  • Language:
  • Keywords:
    Clinical Trials as Topic, Dependovirus, Drug Evaluation, Preclinical, Genetic Therapy, Genetic Vectors, Hemophilia B, Humans
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 these issues. However, until now, clinical trials using gene therapy to treat hemophilia have failed to demonstrate sustained efficacy, although a vector based on a self-complementary adeno-associated virus has recently shown promise. This article will briefly outline a novel gene-transfer approach using self-complementary adeno-associated viral vectors using hemophilia B as a target disorder. This approach is currently being evaluated in the clinic. We will provide an overview of the development of self-complementary adeno-associated virus vectors as well as preclinical and clinical data with this vector system.
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