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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A George, Lindsey A.; Monahan, Paul E.; Eyster, M. Elaine; Sullivan, Spencer K.; Ragni, Margaret, V; Croteau, Stacy E.; Rasko, John E. J.; Recht, Michael; Samelson-Jones, Benjamin J.; MacDougall, Amy; Jaworski, Kristen; Noble, Robert; Curran, Marla; Kuranda, Klaudia; Mingozzi, Federico; Chang, Tiffany; Reape, Kathleen Z.; Anguela, Xavier M.; High, Katherine A. Share Save
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease Phase 3 Results at 3 and 4 Years Maguire, Albert M.; Russell, Stephen; Chung, Daniel C.; Yu, Zi-Fan; Tillman, Amy; Drack, Arlene V.; Simonelli, Francesca; Leroy, Bart P.; Reape, Kathleen Z.; High, Katherine A.; Bennett, Jean Share Save
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua Robinson, Mary M.; George, Lindsey A.; Carr, Marcus E.; Samelson-Jones, Benjamin J.; Arruda, Valder R.; Murphy, John E.; Rybin, Denis; Rupon, Jeremy; High, Katherine A.; Tiefenbacher, Stefan Share Save
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Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B George, Lindsey A.; Ragni, Margaret, V; Rasko, John E. J.; Raffini, Leslie J.; Samelson-Jones, Benjamin J.; Ozelo, Margareth; Hazbon, Maria; Runowski, Alexa R.; Wellman, Jennifer A.; Wachtel, Katie; Chen, Yifeng; Anguela, Xavier M.; Kuranda, Klaudia; Mingozzi, Federico; High, Katherine A. Share Save
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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy Results of Phase 1 and 3 Trials Maguire, Albert M.; Russell, Stephen; Wellman, Jennifer A.; Chung, Daniel C.; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan; Marshall, Kathleen A.; McCague, Sarah; Reichert, Hannah; Davis, Maria; Simonelli, Francesca; Leroy, Bart P.; Wright, J. Fraser; High, Katherine A.; Bennett, Jean Share Save
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The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene Chung, Daniel C.; Bertelsen, Mette; Lorenz, Birgit; Pennesi, Mark E.; Leroy, Bart P.; Hamel, Christian P.; Pierce, Eric; Sallum, Juliana; Larsen, Michael; Stieger, Knut; Preising, Markus; Weleber, Richard; Yang, Paul; Place, Emily; Liu, Emily; Schaefer, Grace; Distefano-Pappas, Julie; Elci, Okan U.; Mccague, Sarah; Wellman, Jennifer A.; High, Katherine A.; Reape, Kathleen Z. Share Save
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Hepato-entrained B220+CD11c+NK1.1+ cells regulate pre-metastatic niche formation in thelung Hiratsuka, Sachie; Tomita, Takeshi; Mishima, Taishi; Matsunaga, Yuta; Omori, Tsutomu; Ishibashi, Sachie; Yamaguchi, Satoshi; Hosogane, Tsuyoshi; Watarai, Hiroshi; Omori-Miyake, Miyuki; Yamamoto, Tomoko; Shibata, Noriyuki; Watanabe, Akira; Aburatani, Hiroyuki; Tomura, Michio; High, Katherine A.; Maru, Yoshiro Share Save
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant George, L. A.; Sullivan, S. K.; Giermasz, A.; Rasko, J. E. J.; Samelson-Jones, B. J.; Ducore, J.; Cuker, A.; Sullivan, L. M.; Majumdar, S.; Teitel, J.; McGuinn, C. E.; Ragni, M. V.; Luk, A. Y.; Hui, D.; Wright, J. F.; Chen, Y.; Liu, Y.; Wachtel, K.; Winters, A.; Tiefenbacher, S.; Arruda, V. R.; van der Loo, J. C. M.; Zelenaia, O.; Takefman, D.; Carr, M. E.; Couto, L. B.; Anguela, X. M.; High, K. A. Share Save
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies Chung, Daniel C.; McCague, Sarah; Yu, Zi-Fan; Thill, Satha; DiStefano-Pappas, Julie; Bennett, Jean; Cross, Dominique; Marshall, Kathleen; Wellman, Jennifer; High, Katherine A. Share Save
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial Russell, Stephen; Bennett, Jean; Wellman, Jennifer A.; Chung, Daniel C.; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan; McCague, Sarah; Cross, Dominique; Marshall, Kathleen A.; Walshire, Jean; Kehoe, Taylor L.; Reichert, Hannah; Davis, Maria; Raffini, Leslie; George, Lindsey A.; Hudson, F. Parker; Dingfield, Laura; Zhu, Xiaosong; Haller, Julia A.; Sohn, Elliott H.; Mahajan, Vinit B.; Pfeifer, Wanda; Weckmann, Michelle; Johnson, Chris; Gewaily, Dina; Drack, Arlene; Stone, Edwin; Wachtel, Katie; Simonelli, Francesca; Leroy, Bart P.; Wright, J. Fraser; High, Katherine A.; Maguire, Albert M. Share Save
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