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Gene therapies for hemophilia hit the mark in clinical trials

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Two recent studies describe clinical successes for single-dose gene therapy in trials for two forms of hemophilia.

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Figure 1: Gene therapy strategies for hemophilia.

Marina Corral Spence/Springer Nature

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Authors and Affiliations

  1. Department of Biomedical Engineering and the Center for Genomic and Computational Biology, Duke University, Durham, North Carolina, USA

    Adrian K Pickar & Charles A Gersbach

Authors
  1. Adrian K Pickar
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  2. Charles A Gersbach
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Corresponding author

Correspondence to Charles A Gersbach.

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Competing interests

A.K.P. and C.A.G. are inventors on patent applications related to gene therapy. C.A.G. is a scientific advisor to Sarepta Therapeutics, Locus Biosciences and Element Genomics.

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Pickar, A., Gersbach, C. Gene therapies for hemophilia hit the mark in clinical trials. Nat Med 24, 121–122 (2018). https://doi.org/10.1038/nm.4492

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