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Journal of Medical Genetics
|
September 12, 2006
Development of a genotyping microarray for Usher syndrome
Frans P M Cremers, William J Kimberling, Maigi Külm, et al.
Lancet (London, England)
|
October 27, 2009
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M Maguire, Katherine A High, Alberto Auricchio, et al.
Human Mutation
|
July 22, 2008
Identification of 34 novel and 56 known FOXL2 mutations in patients with Blepharophimosis syndrome
Diane Beysen, Sarah De Jaegere, David Amor, et al.
Lancet (London, England)
|
July 18, 2017
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 Russell, Jean Bennett, Jennifer A Wellman, et al.
Nature Medicine
|
April 5, 2022
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Stephen R Russell, Arlene V Drack, Artur V Cideciyan, et al.
American Journal of Human Genetics
|
October 15, 2022
Multi-omics approach dissects cis-regulatory mechanisms underlying North Carolina macular dystrophy, a retinal enhanceropathy
Stijn Van de Sompele, Kent W Small, Munevver Burcu Cicekdal, et al.
Ophthalmology. Retina
|
March 22, 2022
The Natural History of Leber Congenital Amaurosis and Cone-Rod Dystrophy Associated with Variants in the GUCY2D Gene
Leo C Hahn, Michalis Georgiou, Hind Almushattat, et al.
Translational Vision Science & Technology
|
August 25, 2020
Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium
Debra A Thompson, Alessandro Iannaccone, Robin R Ali, et al.
Research Square
|
May 19, 2025
RPE65 variant p.(E519K) causes a novel dominant adult-onset maculopathy in 83 affected individuals
Eline Van Vooren, Filip Van den Broeck, Quinten Mahieu, et al.
Investigative Ophthalmology & Visual Science
|
September 23, 2025
RPE65 Variant p.(E519K) Causes a Novel Dominant Adult-Onset Maculopathy in 83 Affected Individuals
Eline Van Vooren, Filip Van Den Broeck, Quinten Mahieu, et al.
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of 44
Search research articles
Search
Showing results (421-430 of 438) with videos related to
Sort By:
Page
of 44
Journal of Medical Genetics
|
September 12, 2006
Development of a genotyping microarray for Usher syndrome
Frans P M Cremers, William J Kimberling, Maigi Külm, et al.
Lancet (London, England)
|
October 27, 2009
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M Maguire, Katherine A High, Alberto Auricchio, et al.
Human Mutation
|
July 22, 2008
Identification of 34 novel and 56 known FOXL2 mutations in patients with Blepharophimosis syndrome
Diane Beysen, Sarah De Jaegere, David Amor, et al.
Lancet (London, England)
|
July 18, 2017
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 Russell, Jean Bennett, Jennifer A Wellman, et al.
Nature Medicine
|
April 5, 2022
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Stephen R Russell, Arlene V Drack, Artur V Cideciyan, et al.
American Journal of Human Genetics
|
October 15, 2022
Multi-omics approach dissects cis-regulatory mechanisms underlying North Carolina macular dystrophy, a retinal enhanceropathy
Stijn Van de Sompele, Kent W Small, Munevver Burcu Cicekdal, et al.
Ophthalmology. Retina
|
March 22, 2022
The Natural History of Leber Congenital Amaurosis and Cone-Rod Dystrophy Associated with Variants in the GUCY2D Gene
Leo C Hahn, Michalis Georgiou, Hind Almushattat, et al.
Translational Vision Science & Technology
|
August 25, 2020
Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium
Debra A Thompson, Alessandro Iannaccone, Robin R Ali, et al.
Research Square
|
May 19, 2025
RPE65 variant p.(E519K) causes a novel dominant adult-onset maculopathy in 83 affected individuals
Eline Van Vooren, Filip Van den Broeck, Quinten Mahieu, et al.
Investigative Ophthalmology & Visual Science
|
September 23, 2025
RPE65 Variant p.(E519K) Causes a Novel Dominant Adult-Onset Maculopathy in 83 Affected Individuals
Eline Van Vooren, Filip Van Den Broeck, Quinten Mahieu, et al.
Page
of 44