Albert M. Maguire
University of Pennsylvania
220 Papers
1.6K Citations
Albert M. Maguire is an academic researcher from University of Pennsylvania. The author has contributed to research in topics: Medicine & Retina. The author has an hindex of 61, co-authored 207 publications. Previous affiliations of Albert M. Maguire include Children's Hospital of Philadelphia & Scheie Eye Institute.
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Papers
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire,Francesca Simonelli,Eric A. Pierce,Edward N. Pugh,Federico Mingozzi,Jeannette L. Bennicelli,Sandro Banfi,Kathleen A. Marshall,Francesco Testa,Enrico Maria Surace,Settimio Rossi,Arkady Lyubarsky,Valder R. Arruda,Barbara A. Konkle,Edwin M. Stone,Edwin M. Stone,Junwei Sun,Jonathan B. Jacobs,L. F. Dell'Osso,Richard W. Hertle,Jian Xing Ma,T. Michael Redmond,Xiaosong Zhu,Bernd Hauck,Olga Zelenaia,Kenneth S. Shindler,Maureen G. Maguire,J. Fraser Wright,Nicholas J. Volpe,Jennifer Wellman McDonnell,Alberto Auricchio,Katherine A. High,Katherine A. High,Jean Bennett +33 more
TL;DR: This study investigated the safety of subretinal delivery of a recombinant adeno-associated virus (AAV) carrying RPE65 complementary DNA (cDNA) and found three patients with LCA2 had an acceptable local and systemic adverse-event profile after delivery of AAV2.hRPE65v2.
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,Daniel C. Chung,Zi Fan Yu,Amy Tillman,Janet Wittes,Julie Pappas,Okan U. Elci,Sarah McCague,Dominique Cross,Kathleen A. Marshall,Jean Walshire,Taylor Kehoe,Hannah Reichert,Maria C. Davis,Leslie Raffini,Lindsey A. George,F. Parker Hudson,Laura E. Dingfield,Xiaosong Zhu,Julia A. Haller,Elliott H. Sohn,Vinit B. Mahajan,Wanda Pfeifer,Michelle T. Weckmann,Chris A. Johnson,Dina Y. Gewaily,Arlene V. Drack,Edwin M. Stone,Katie Wachtel,Francesca Simonelli,Bart P. Leroy,Bart P. Leroy,J. Fraser Wright,Katherine A. High,Albert M. Maguire +36 more
TL;DR: Voretigene neparvovec gene replacement improved functional vision in RPE65-mediated inherited retinal dystrophy previously medically untreatable.
1.5K
Gene therapy restores vision in a canine model of childhood blindness.
Gregory M. Acland,Gustavo D. Aguirre,Jharna Ray,Qi Zhang,Tomas S. Aleman,Artur V. Cideciyan,Susan E. Pearce-Kelling,Vibha Anand,Yong Zeng,Albert M. Maguire,Samuel G. Jacobson,William W. Hauswirth,Jean Bennett +12 more
TL;DR: The results indicate that visual function was restored in this large animal model of childhood blindness, and gene therapy directed at photoreceptors and RPE in a large-animal model of human disease has not been reported.
1.2K
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M. Maguire,Albert M. Maguire,Katherine A. High,Katherine A. High,Alberto Auricchio,J. Fraser Wright,J. Fraser Wright,Eric A. Pierce,Eric A. Pierce,Francesco Testa,Federico Mingozzi,Jeannette L. Bennicelli,Gui-Shuang Ying,Settimio Rossi,Ann Fulton,Kathleen A. Marshall,Sandro Banfi,Daniel C. Chung,Jessica I. W. Morgan,Bernd Hauck,Olga Zelenaia,Xiaosong Zhu,Leslie Raffini,Frauke Coppieters,Elfride De Baere,Kenneth S. Shindler,Nicholas J. Volpe,Enrico Maria Surace,Carmela Acerra,Arkady Lyubarsky,T. Michael Redmond,T. Michael Redmond,Edwin M. Stone,Edwin M. Stone,Junwei Sun,Jenni Fer Uvellman Mcdonnell,Bart P. Leroy,Bart P. Leroy,Francesca Simonelli,Jean Bennett,Jean Bennett +40 more
TL;DR: The safety, extent, and stability of improvement in vision in all patients support the use of AAV-mediated gene therapy for treatment of inherited retinal diseases, with early intervention resulting in the best potential gain.
908
•Journal Article
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model.
Samuel Jotham Reich,Joshua Fosnot,A. Kuroki,Waixing Tang,Xiangyang Yang,Albert M. Maguire,Jean Bennett,Michael J. Tolentino +7 more
TL;DR: Delivery of siRNA can be used in vitro and in vivo to target specific RNAs and to reduce the levels of the specific protein product in the targeted cells, suggesting that RNA interference has potential for application to studies of retinal biology and for the treatment of a variety of retina diseases, including those involving abnormal blood vessel growth.