J
Jennifer Wellman
Researcher at Children's Hospital of Philadelphia
Publications - 22
Citations - 2835
Jennifer Wellman is an academic researcher from Children's Hospital of Philadelphia. The author has contributed to research in topics: Capsid & Adeno-associated virus. The author has an hindex of 11, co-authored 21 publications receiving 2118 citations.
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
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.
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
Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
Francesco Testa,Albert M. Maguire,Albert M. Maguire,Settimio Rossi,Eric A. Pierce,Paolo Melillo,Kathleen A. Marshall,Sandro Banfi,Enrico Maria Surace,Junwei Sun,Carmela Acerra,J. Fraser Wright,J. Fraser Wright,Jennifer Wellman,Katherine A. High,Katherine A. High,Alberto Auricchio,Jean Bennett,Jean Bennett,Francesca Simonelli +19 more
TL;DR: The long-term follow-up data on the 5-patient Italian cohort involved in the LCA2 gene therapy clinical trial clearly showed a stability of improvement in visual and retinal function that had been achieved a few months after treatment.
Journal ArticleDOI
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Albert M. Maguire,Albert M. Maguire,Stephen R. Russell,Jennifer Wellman,Daniel C. Chung,Zi-Fan Yu,Amy Tillman,Janet Wittes,Julie Pappas,Okan U. Elci,Kathleen A. Marshall,Sarah McCague,Hannah Reichert,Maria C. Davis,Francesca Simonelli,Bart P. Leroy,J. Fraser Wright,Katherine A. High,Jean Bennett +18 more
TL;DR: After VN gene augmentation therapy, there was a favorable benefit-to-risk profile with similar improvement demonstrated in navigational ability and light sensitivity among 3 groups of subjects with RPE65 mutation-associated IRD, a degenerative disease that progresses to complete blindness.