Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

@article{Nathwani2006SelfcomplementaryAV,
  title={Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.},
  author={Amit Chunilal Nathwani and John Mark Nicholas Timm Gray and Catherine Y. C. Ng and Junfang Zhou and Yunyu Spence and Simon N Waddington and Edward G. D. Tuddenham and Geoffrey Kemball-Cook and Jenny Mcintosh and Mariette Boon-Spijker and Koen Mertens and Andrew M. Davidoff},
  journal={Blood},
  year={2006},
  volume={107 7},
  pages={2653-61}
}
Transduction with recombinant adeno-associated virus (AAV) vectors is limited by the need to convert its single-stranded (ss) genome to transcriptionally active double-stranded (ds) forms. For AAV-mediated hemophilia B (HB) gene therapy, we have overcome this obstacle by constructing a liver-restricted mini-human factor IX (hFIX) expression cassette that can be packaged as complementary dimers within individual AAV particles. Molecular analysis of murine liver transduced with these self… CONTINUE READING
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Adenoassociated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome 2660 NATHWANI et al BLOOD, 1 APRIL

  • DM McCarty, H Fu, PE Monahan
  • For personal use only. on April
  • 2006

Human immune responses to AAV - 2 capsid may limit duration of expression in liver - directed gene transfer in humans with hemophilia B [ abstract ]

  • K High, M Tigges, CS Manno
  • Blood
  • 2004

Human immune responses to AAV-2 capsid may limit duration of expression in liver-directed gene transfer in humans with hemophilia

  • K High, M Tigges, CS Manno
  • B [abstract]. Blood
  • 2004

Immune response to AAV and to factor IX in a phase I study of AAV-mediated liver directed gene transfer for hemophilia B [abstract

  • K High, CS Manno, DE Sabatino
  • Mol Ther
  • 2004

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