Search Results - "Wellman, Jennifer A."

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B by 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.

“…Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene…”
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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 by 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

“…To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral vector-based gene therapy for RPE65 mutation-associated inherited retinal…”
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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial by 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

“…Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and…”
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The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene by 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.

“…To delineate the natural history of visual parameters over time in individuals with biallelic RPE65 mutation–associated inherited retinal dystrophy (IRD);…”
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Genetic Medicine for Hearing Loss: OTOF as Exemplar by Hickox, Ann E, Valero, Michelle D, McLaughlin, James T, Robinson, Gregory S, Wellman, Jennifer A, McKenna, Michael J, Sewell, William F, Simons, Emmanuel J

“…Millions of people worldwide have disabling hearing loss because one of their genes generates an incorrect version of some specific protein the ear requires…”
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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy by 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

“…To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral vector–based gene therapy for RPE65 mutation–associated inherited retinal…”
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Year 3 results and age-stratified analyses for a phase 3 trial of voretigene neparvovec in RPE65 mutation-associated inherited retinal disease by Drack, Arlene V., Bennett, Jean, Russell, Stephen, Wellman, Jennifer A., Chung, Daniel C., High, Katherine A., Yu, Zi-Fan, Maguire, Albert M.

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