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Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia Aleman, Tomas S.; Huckfeldt, Rachel M.; Serrano, Leona W.; Pearson, Denise J.; Vergilio, Grace K.; McCague, Sarah; Marshall, Kathleen A.; Ashtari, Manzar; Doan, Tu M.; Weigel-DiFranco, Carol A.; Biron, Bethany S.; Wen, Xiao-Hong; Chung, Daniel C.; Liu, Emily; Ferenchak, Kevin; Morgan, Jessica I. W.; Pierce, Eric A.; Eliott, Dean; Bennett, Jean; Comander, Jason; Maguire, Albert M. 分享 收藏
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 分享 收藏
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Treatment Potential for LCA5-Associated Leber Congenital Amaurosis Uyhazi, Katherine E.; Aravand, Puya; Bell, Brent A.; Wei, Zhangyong; Leo, Lanfranco; Serrano, Leona W.; Pearson, Denise J.; Shpylchak, Ivan; Pham, Jennifer; Vasireddy, Vidyullatha; Bennett, Jean; Aleman, Tomas S. 分享 收藏
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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 分享 收藏
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Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1-9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons Duong, Thu T.; Lim, James; Vasireddy, Vidyullatha; Papp, Tyler; Hung Nguyen; Leo, Lanfranco; Pan, Jieyan; Zhou, Shangzhen; Chen, H. Isaac; Bennett, Jean; Mills, Jason A. 分享 收藏
An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications (vol 5, 17105, 2015) Sochor, Matthew A.; Vasireddy, Vidyullatha; Drivas, Theodore G.; Wojno, Adam; Duong, Thu T.; Shpylchak, Ivan; Bennicelli, Jeannette; Chung, Daniel; Bennett, Jean; Lewis, Mitchell 分享 收藏