Zobrazeno 1 - 10
of 27
pro vyhledávání: '"Sarah McCague"'
Autor:
Tomas S. Aleman, Rachel M. Huckfeldt, Leona W. Serrano, Denise J. Pearson, Grace K. Vergilio, Sarah McCague, Kathleen A. Marshall, Manzar Ashtari, Tu M. Doan, Carol A. Weigel-DiFranco, Bethany S. Biron, Xiao-Hong Wen, Daniel C. Chung, Emily Liu, Kevin Ferenchak, Jessica I.W. Morgan, Eric A. Pierce, Dean Eliott, Jean Bennett, Jason Comander, Albert M. Maguire
Publikováno v:
Ophthalmology. 129:1177-1191
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::7658d17b9d4a9fa041bddb7999ff239e
https://doi.org/10.1016/j.ophtha.2022.06.006
https://doi.org/10.1016/j.ophtha.2022.06.006
Autor:
Tomas S, Aleman, Rachel M, Huckfeldt, Leona W, Serrano, Denise J, Pearson, Grace K, Vergilio, Sarah, McCague, Kathleen A, Marshall, Manzar, Ashtari, Tu M, Doan, Carol A, Weigel-DiFranco, Bethany S, Biron, Xiao-Hong, Wen, Daniel C, Chung, Emily, Liu, Kevin, Ferenchak, Jessica I W, Morgan, Eric A, Pierce, Dean, Eliott, Jean, Bennett, Jason, Comander, Albert M, Maguire
Publikováno v:
Ophthalmology. 129(10)
To assess the safety of the subretinal delivery of a recombinant adeno-associated virus serotype 2 (AAV2) vector carrying a human choroideremia (CHM)-encoding cDNA in CHM.Prospective, open-label, nonrandomized, dose-escalation, phase I/II clinical tr
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::175d44fa052beacd88edf6041f266d41
https://pubmed.ncbi.nlm.nih.gov/35714735
https://pubmed.ncbi.nlm.nih.gov/35714735
Autor:
Amy Tillman, Kathleen A. Marshall, Maria C. Davis, Janet Wittes, J. Fraser Wright, Hannah Reichert, Bart P. Leroy, Julie Pappas, Jean Bennett, Zi-Fan Yu, Stephen R. Russell, Jennifer Wellman, Okan U. Elci, Katherine A. High, Albert M. Maguire, Sarah McCague, Francesca Simonelli, Daniel C. Chung
Publikováno v:
Ophthalmology. 126:1273-1285
Purpose To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral vector–based gene therapy for RPE65 mutation–associated inherited retinal dystrophy (IRD), including results of a phase 1 follow-on study at year 4 and pha
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::f1a8befd946bae466b51122c3ffb3915
https://doi.org/10.1016/j.ophtha.2019.06.017
https://doi.org/10.1016/j.ophtha.2019.06.017
Autor:
Dominique Cross, Kathleen A. Marshall, Sarah McCague, Katherine A. High, Satha Thill, Daniel C. Chung, Zi-Fan Yu, Jennifer Wellman, Julie DiStefano-Pappas, Jean Bennett
Publikováno v:
Clinical & Experimental Ophthalmology. 46:247-259
Importance This novel endpoint tracks functional vision changes in patients with inherited retinal dystrophies (IRDs) over time. Background The aims of the study were to determine whether a multi-luminance mobility test (MLMT) can detect functional v
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::097517649c7c55a8bb5cb8a9a09464c7
https://doi.org/10.1111/ceo.13022
https://doi.org/10.1111/ceo.13022
Autor:
Xiaosong Zhu, Jean Bennett, Chris A. Johnson, Katie Wachtel, Jean Walshire, Zi Fan Yu, Julie Pappas, Taylor Kehoe, Elliott H. Sohn, Jennifer Wellman, Vinit B. Mahajan, Bart P. Leroy, Michelle T. Weckmann, Francesca Simonelli, Okan U. Elci, Dominique Cross, Amy Tillman, J. Fraser Wright, Lindsey A. George, Kathleen A. Marshall, Julia A. Haller, Maria C. Davis, Edwin M. Stone, Janet Wittes, Laura E. Dingfield, Dina Y. Gewaily, Sarah McCague, Leslie Raffini, Katherine A. High, Albert M. Maguire, Hannah Reichert, Arlene V. Drack, Daniel C. Chung, Wanda Pfeifer, F. Parker Hudson, Stephen R. Russell
Publikováno v:
The Lancet. 390:849-860
Background Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4f182894740f3f23311e678456a122ca
https://doi.org/10.1016/s0140-6736(17)31868-8
https://doi.org/10.1016/s0140-6736(17)31868-8
Autor:
Juliana Maria Ferraz Sallum, Michael Larsen, Julie DiStefano-Pappas, Kathleen Z Reape, Sarah McCague, Eric A. Pierce, Emily Place, Mette Bertelsen, Daniel C. Chung, Markus N. Preising, Mark E. Pennesi, Jennifer Wellman, Grace Schaefer, Katherine A. High, Bart P. Leroy, Richard G. Weleber, Okan U. Elci, Birgit Lorenz, Emily Liu, Christian P. Hamel, Paul Yang, Knut Stieger
Publikováno v:
American Journal of Ophthalmology
American Journal of Ophthalmology, Elsevier Masson, 2019, 199, pp.58-70. ⟨10.1016/j.ajo.2018.09.024⟩
American Journal of Ophthalmology, Elsevier Masson, 2019, 199, pp.58-70. ⟨10.1016/j.ajo.2018.09.024⟩
International audience; PURPOSE:To delineate the natural history of visual parameters over time in individuals with biallelic RPE65 mutation-associated inherited retinal dystrophy (IRD); describe the range of causative mutations; determine potential
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::30db5142d55e58da94fe2de3001ddb44
https://hal.umontpellier.fr/hal-02558148
https://hal.umontpellier.fr/hal-02558148
Autor:
Albert M, Maguire, Stephen, Russell, Jennifer A, Wellman, Daniel C, Chung, Zi-Fan, Yu, Amy, Tillman, Janet, Wittes, Julie, Pappas, Okan, Elci, Kathleen A, Marshall, Sarah, McCague, Hannah, Reichert, Maria, Davis, Francesca, Simonelli, Bart P, Leroy, J Fraser, Wright, Katherine A, High, Jean, Bennett
Publikováno v:
Ophthalmology
To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral vector-based gene therapy for RPE65 mutation-associated inherited retinal dystrophy (IRD), including results of a phase 1 follow-on study at year 4 and phase 3 study a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::872301ff0febc7bab7835c6fe8dc0d40
https://pubmed.ncbi.nlm.nih.gov/31443790
https://pubmed.ncbi.nlm.nih.gov/31443790
Autor:
Daniel C, Chung, Sarah, McCague, Zi-Fan, Yu, Satha, Thill, Julie, DiStefano-Pappas, Jean, Bennett, Dominique, Cross, Kathleen, Marshall, Jennifer, Wellman, Katherine A, High
Publikováno v:
Clinicalexperimental ophthalmology. 46(3)
IMPORTANCE: This novel endpoint tracks functional vision changes in patients with inherited retinal dystrophies (IRDs) over time. BACKGROUND: The aims of the study were to determine whether a multi-luminance mobility test (MLMT) can detect functional
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::df6c8edc2d7b07c80531f8cac1a6f45a
https://pubmed.ncbi.nlm.nih.gov/28697537
https://pubmed.ncbi.nlm.nih.gov/28697537
Autor:
Alberto Auricchio, Katherine A. High, Julie DiStefano-Pappas, Kathleen A. Marshall, Dominique Cross, J. Fraser Wright, Albert M. Maguire, Puya Aravand, Okan U. Elci, Sarah McCague, Federico Mingozzi, Laura Cyckowski, Bart P. Leroy, Daniel C. Chung, Jennifer Wellman, Jason Ruggiero, Francesca Simonelli, Eric A. Pierce, Junwei Sun, Manzar Ashtari, Jeannette L. Bennicelli, Jean Bennett
Publikováno v:
LANCET
LANCET, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩
The Lancet
The Lancet, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩
LANCET, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩
The Lancet
The Lancet, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩
International audience; BACKGROUND: Safety and efficacy have been shown in a phase 1 dose-escalation study involving a unilateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in i
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f9adfa71eeaec8bb5fe1e821d109f2c2
http://hdl.handle.net/11591/358419
http://hdl.handle.net/11591/358419
Autor:
Tuohy, Gearóid P.1 (AUTHOR) Roly.Megaw@ed.ac.uk, Megaw, Roly1,2 (AUTHOR), Dal Monte, Massimo (AUTHOR), Khanna, Hemant (AUTHOR)
Publikováno v:
Biomolecules (2218-273X). May2021, Vol. 11 Issue 5, p760. 1p.