Concepedia
Statistics
Publications
5
Citations
2.9K
H-Index
Active since
2016
Affiliations
University of Pennsylvania(Current)
Spark Therapeutics (United States)
Children's Hospital of Philadelphia
University of Pittsburgh
University of California Davis Medical Center
Katie Wachtel is an author at University of Pennsylvania specializing in medicine, hematology, and gene therapy.
Publications per year
2016–2020
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen R. Russell, Jean Bennett, Jennifer Wellman et al. · The Lancet · 2017 · 1.9K citations
Medicine, Ophthalmology, Vision Science +14
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
Lindsey A. George, Spencer K. Sullivan, Adam Giermasz et al. · New England Journal of Medicine · 2017 · 759 citations · Full text
Medicine, Hematology, Molecular Medicine +15
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Lindsey A. George, Margaret V. Ragni, John E.J. Rasko et al. · Molecular Therapy · 2020 · 209 citations · Full text
Medicine, Hematology, Immunology +13
A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
Katherine A. High, Lindsey A. George, M. Elaine Eyster et al. · Blood · 2018 · 60 citations
Medicine, Hematology, Immunology +15
Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression
Lindsey A. George, Spencer K. Sullivan, Adam Giermasz et al. · Blood · 2016 · 19 citations
Life Sciences, Hematology, Molecular Biology +11
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