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Human Gene Therapy
|
March 28, 2018
Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells
Anai Gonzalez-Cordero, Debbie Goh, Kamil Kruczek, et al.
The New England Journal of Medicine
|
April 29, 2008
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge, Alexander J Smith, Susie S Barker, et al.
Plos One
|
March 14, 2012
Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy
Mei Hong Tan, Donna S Mackay, Jill Cowing, et al.
Human Gene Therapy
|
December 29, 2025
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration
Wenfei Liu, Amy F Geard, Giulia Massaro, et al.
Cell Reports
|
April 21, 2021
Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors
Joana Ribeiro, Christopher A Procyk, Emma L West, et al.
Stem Cells (Dayton, Ohio)
|
May 4, 2026
Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration
Christopher A Procyk, Anna Melati, Menahil Tariq, et al.
Ophthalmology
|
June 10, 2018
Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in Macular Degeneration
Manjit S Mehat, Venki Sundaram, Caterina Ripamonti, et al.
Stem Cell Reports
|
August 29, 2017
Recapitulation of Human Retinal Development from Human Pluripotent Stem Cells Generates Transplantable Populations of Cone Photoreceptors
Anai Gonzalez-Cordero, Kamil Kruczek, Arifa Naeem, et al.
American Journal of Ophthalmology
|
May 12, 2023
First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia
Michel Michaelides, Nashila Hirji, Sui Chien Wong, et al.
Lancet (London, England)
|
February 22, 2025
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Michel Michaelides, Yannik Laich, Sui Chien Wong, et al.
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Search research articles
Search
Showing results (101-110 of 113) with videos related to
Sort By:
Page
of 12
Human Gene Therapy
|
March 28, 2018
Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells
Anai Gonzalez-Cordero, Debbie Goh, Kamil Kruczek, et al.
The New England Journal of Medicine
|
April 29, 2008
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge, Alexander J Smith, Susie S Barker, et al.
Plos One
|
March 14, 2012
Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy
Mei Hong Tan, Donna S Mackay, Jill Cowing, et al.
Human Gene Therapy
|
December 29, 2025
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration
Wenfei Liu, Amy F Geard, Giulia Massaro, et al.
Cell Reports
|
April 21, 2021
Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors
Joana Ribeiro, Christopher A Procyk, Emma L West, et al.
Stem Cells (Dayton, Ohio)
|
May 4, 2026
Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration
Christopher A Procyk, Anna Melati, Menahil Tariq, et al.
Ophthalmology
|
June 10, 2018
Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in Macular Degeneration
Manjit S Mehat, Venki Sundaram, Caterina Ripamonti, et al.
Stem Cell Reports
|
August 29, 2017
Recapitulation of Human Retinal Development from Human Pluripotent Stem Cells Generates Transplantable Populations of Cone Photoreceptors
Anai Gonzalez-Cordero, Kamil Kruczek, Arifa Naeem, et al.
American Journal of Ophthalmology
|
May 12, 2023
First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia
Michel Michaelides, Nashila Hirji, Sui Chien Wong, et al.
Lancet (London, England)
|
February 22, 2025
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Michel Michaelides, Yannik Laich, Sui Chien Wong, et al.
Page
of 12