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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 23, 2008
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
Jeannette Bennicelli, John Fraser Wright, Andras Komaromy, et al.
Science Translational Medicine
|
February 11, 2012
AAV2 gene therapy readministration in three adults with congenital blindness
Jean Bennett, Manzar Ashtari, Jennifer Wellman, et al.
The Journal of Clinical Investigation
|
May 25, 2011
Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice
Karsten Boldt, Dorus A Mans, Jungyeon Won, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 3, 2009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
Francesca Simonelli, Albert M Maguire, Francesco Testa, et al.
Ophthalmology
|
June 17, 2022
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial
Tomas S Aleman, Rachel M Huckfeldt, Leona W Serrano, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Lancet (London, England)
|
July 5, 2016
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, Jennifer Wellman, Kathleen A Marshall, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 2, 2025
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial
Tomas S Aleman, Katherine E Uyhazi, Alejandro J Roman, et al.
Investigative Ophthalmology & Visual Science
|
November 15, 2012
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease
Kari Branham, Mohammad Othman, Matthew Brumm, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 21, 2018
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness
Ji Yun Song, Puya Aravand, Sergei Nikonov, et al.
Page
of 18
Search research articles
Search
Showing results (161-170 of 175) with videos related to
Sort By:
Page
of 18
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 23, 2008
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
Jeannette Bennicelli, John Fraser Wright, Andras Komaromy, et al.
Science Translational Medicine
|
February 11, 2012
AAV2 gene therapy readministration in three adults with congenital blindness
Jean Bennett, Manzar Ashtari, Jennifer Wellman, et al.
The Journal of Clinical Investigation
|
May 25, 2011
Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice
Karsten Boldt, Dorus A Mans, Jungyeon Won, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 3, 2009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
Francesca Simonelli, Albert M Maguire, Francesco Testa, et al.
Ophthalmology
|
June 17, 2022
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial
Tomas S Aleman, Rachel M Huckfeldt, Leona W Serrano, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Lancet (London, England)
|
July 5, 2016
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, Jennifer Wellman, Kathleen A Marshall, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 2, 2025
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial
Tomas S Aleman, Katherine E Uyhazi, Alejandro J Roman, et al.
Investigative Ophthalmology & Visual Science
|
November 15, 2012
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease
Kari Branham, Mohammad Othman, Matthew Brumm, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 21, 2018
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness
Ji Yun Song, Puya Aravand, Sergei Nikonov, et al.
Page
of 18