B
Bart P. Leroy
Researcher at Children's Hospital of Philadelphia
Publications - 157
Citations - 7988
Bart P. Leroy is an academic researcher from Children's Hospital of Philadelphia. The author has contributed to research in topics: Retinitis pigmentosa & Visual acuity. The author has an hindex of 40, co-authored 139 publications receiving 6460 citations. Previous affiliations of Bart P. Leroy include Ghent University Hospital & Ghent University.
Papers
More filters
Journal ArticleDOI
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.
Journal ArticleDOI
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.
Journal ArticleDOI
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
Jean Bennett,Jean Bennett,Jennifer Wellman,Kathleen A. Marshall,Sarah McCague,Manzar Ashtari,Manzar Ashtari,Julie DiStefano-Pappas,Okan U. Elci,Daniel C. Chung,Junwei Sun,Junwei Sun,J. Fraser Wright,Dominique Cross,Puya Aravand,Laura Cyckowski,Jeannette L. Bennicelli,Federico Mingozzi,Alberto Auricchio,Eric A. Pierce,Jason Ruggiero,Bart P. Leroy,Bart P. Leroy,Francesca Simonelli,Katherine A. High,Katherine A. High,Albert M. Maguire +26 more
TL;DR: To the knowledge, AAV2-hRPE65v2 is the first successful gene therapy administered to the contralateral eye and the results highlight the use of several outcome measures and help to delineate the variables that contribute to maximal benefit from gene augmentation therapy in this disease.
Journal ArticleDOI
The spectrum of ocular phenotypes caused by mutations in the BEST1 gene
Camiel J. F. Boon,B. Jeroen Klevering,Bart P. Leroy,Carel B. Hoyng,Jan E.E. Keunen,Anneke I. den Hollander +5 more
TL;DR: The purpose of this review is to describe this spectrum of phenotypes, which includes Best vitelliform macular dystrophy and adult-onset foveomacular viteLLiform Dystrophy, autosomal dominant vitreoretinochoroidopathy, the MRCS (microcornea, rod-cone dystrohy, cataract, posterior staphyloma) syndrome, and autosomal recessive bestrophinopathy.
Journal ArticleDOI
Biallelic Mutation of BEST1 Causes a Distinct Retinopathy in Humans
Rosemary Burgess,Rosemary Burgess,Ian D. Millar,Bart P. Leroy,Jill E. Urquhart,Jill E. Urquhart,Ian M. Fearon,Elfrida De Baere,Peter de Nully Brown,Anthony G. Robson,Anthony G. Robson,Genevieve A. Wright,Philippe Kestelyn,Graham E. Holder,Graham E. Holder,Andrew R. Webster,Andrew R. Webster,Forbes D C Manson,Forbes D C Manson,Forbes D C Manson,Graeme C.M. Black,Graeme C.M. Black,Graeme C.M. Black +22 more
TL;DR: A distinct retinal disorder, autosomal-recessive bestrophinopathy (ARB), that is consequent upon biallelic mutation in BEST1 and is associated with central visual loss, a characteristic retinopathy, an absent electro-oculogram light rise, and a reduced electroretinogram is described.