F
Francesca Simonelli
Researcher at Seconda Università degli Studi di Napoli
Publications - 223
Citations - 10675
Francesca Simonelli is an academic researcher from Seconda Università degli Studi di Napoli. The author has contributed to research in topics: Medicine & Visual acuity. The author has an hindex of 41, co-authored 189 publications receiving 8946 citations. Previous affiliations of Francesca Simonelli include University of Naples Federico II.
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Journal ArticleDOI
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.
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
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
Francesca Simonelli,Albert M. Maguire,Albert M. Maguire,Francesco Testa,Eric A. Pierce,Federico Mingozzi,Jeannette L. Bennicelli,Settimio Rossi,Kathleen A. Marshall,Sandro Banfi,Enrico Maria Surace,Junwei Sun,T. Michael Redmond,Xiaosong Zhu,Kenneth S. Shindler,Gui-Shuang Ying,Carmela Ziviello,Carmela Acerra,Carmela Acerra,J. Fraser Wright,J. Fraser Wright,Jennifer Wellman McDonnell,Katherine A. High,Katherine A. High,Jean Bennett,Jean Bennett,Alberto Auricchio +26 more
TL;DR: The safety of the intervention and the stability of the improvement in visual and retinal function in these subjects support the use of AAV-mediated gene augmentation therapy for treatment of inherited retinal diseases.
Journal ArticleDOI
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Jean Bennett,Jean Bennett,Manzar Ashtari,Jennifer Wellman,Kathleen A. Marshall,Laura Cyckowski,Daniel C. Chung,Daniel C. Chung,Sarah McCague,Eric A. Pierce,Eric A. Pierce,Yifeng Chen,Jeannette L. Bennicelli,Xiaosong Zhu,Gui-Shuang Ying,Junwei Sun,J. Fraser Wright,Alberto Auricchio,Francesca Simonelli,Kenneth S. Shindler,Federico Mingozzi,Katherine A. High,Katherine A. High,Albert M. Maguire,Albert M. Maguire +24 more
TL;DR: The researchers report that the lack of immune response and the robust safety profile in this readministration gene therapy study may be due in part to the immune-privileged nature of the eye, and the low dose and very pure preparation of the AAV vector.