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September 14, 2022Annual Review of MedicineOpen Access

Adeno-Associated Virus Gene Therapy for Hemophilia

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Authors

BSBenjamin J. Samelson‐JonesChildren's Hospital of PhiladelphiaLGLindsey A. GeorgeChildren's Hospital of Philadelphia

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Samelson‐Jones et al. (2022) studied this question.

synapsesocial.com/papers/69daa9a04a1e15904c8358d5https://doi.org/10.1146/annurev-med-043021-033013
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A2021 · 258 citations
  2. 2Emicizumab Prophylaxis in Hemophilia A with Inhibitors2017 · 1,124 citations
  3. 3Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs2019 · 127 citations
  4. 4Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant2017 · 778 citations
  5. 5Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B2017 · 306 citations