Results 31 to 40 of about 2,056 (145)

RNA-targeting strategies as a platform for ocular gene therapy

, 2023
Genetic medicine is offering hope as new therapies are emerging for many previously untreatable diseases. The eye is at the forefront of these advances, as exemplified by the approval of Luxturna® by the United States Food and Drug Administration (US FDA)
Fry, LE, Hewitt, AW, Chen, FK, Kumar, S, Martin, KR, Liu, G-S, Wang, J-H   +6 more
core   +1 more source

Implementation of experimental cellular (cellular-genetic) therapies on the example of eye diseases

Journal of Education, Health and Sport, 2020
The development of technology and a modern research approach in the 21st century has enabled access to new therapies in many fields of medicine and nanotechnology.
Patrycja Kapczuk, Karolina Rogulska, Anna Stangret, Agata Mularczyk, Angelika Szczęśniak, Katarzyna Topczewska, Konrad Grzeszczak   +6 more
doaj   +1 more source

An Assessment of Genetic Counselors’ Knowledge and Attitudes Regarding Gene Transfer Therapies [PDF]

, 2021
Background: There are currently two FDA approved gene transfer therapies, Luxturna (voretigene neparvovec-rzyl) to treat RPE65 Associated Inherited Retinal Dystrophy and Zolgensma (onasemnogene abeparvovec-xioi) to treat Spinal Muscular Atrophy.
Walsh, Chelsey
core  

The potential of small molecule brain-derived neurotrophic factor: mimetics to treat inherited retinal degeneration [PDF]

, 2019
Is there a need for small molecule neuroprotectants in inherited retinal degeneration (iRDs)? iRDs are a heterogeneous cluster of diseases which lead to blindness in 1 in every 2–3000 people.
Justine Mathoux, Breandán N Kennedy, Andrea Cerquone Perpetuini, Cerquone Perpetuini, Andrea, Mathoux, Justine, Kennedy, Breandán   +5 more
core   +1 more source

Multi-luminance mobility testing after gene therapy in the context of retinal functional diagnostics

, 2023
<jats:title>Abstract</jats:title><jats:sec> <jats:title>Background</jats:title> <jats:p>Voretigene neparvovec (Luxturna®) is the first approved gene therapy for RPE65-linked Leber ...
Katarina Stingl, Kempf, Melanie, Stingl, Krunoslav, Friederike C. Kortüm, Jung, Ronja, Kortüm, Friederike C., Melanie Kempf, Ronja Jung, Saskia Holocher, Stingl, Katarina, Holocher, Saskia, Krunoslav Stingl   +11 more
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Predicting potentially pathogenic effects of hRPE65 missense mutations: a computational strategy based on molecular dynamics simulations

, 2022
The human retinal pigment epithelium-specific 65-kDa protein (hRPE65) plays a crucial role within the retinoid visual cycle and several mutations affecting either its expression level or its enzymatic function are associated with inherited retinal ...
Marco Macchia (409137), Giulio Poli (809754), Ivana Barravecchia (12899701), Alessandro Saba (656433), Gian Carlo Demontis (9774899), Tiziano Tuccinardi (409135), Andrea Sodi (703652), Stanislao Rizzo (4740000)   +7 more
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New Gene Therapy Treatment for Retinitis Pigmentosa

, 2020
Objective: This study was conducted to educate health care professionals on Luxturna (Voretigene neparvovec), a novel FDA approved gene therapy for retinitis pigmentosa, and on identifying and testing procedure for potential candidates for the gene ...
Berezu, Tiana
core   +1 more source

La thérapie génique des rétinites pigmentaires héréditaires

, 2020
International audienceRetinitis pigmentosa is the most common blinding inherited retinal dystrophy. Gene therapy is a burgeoning revolutionary approach that paves the way to treatment of previously incurable diseases.
Jean-Baptiste Ducloyer, Michel Weber, Le Meur, Guylène, Thérèse Cronin, Ducloyer, Jean-Baptiste, Oumeya Adjali, Guylène Le Meur, Cronin, Thérèse, Adjali, Oumeya, Weber, Michel   +9 more
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Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to Date

The Application of Clinical Genetics, 2020
Srikanta Kumar Padhy,1 Brijesh Takkar,2,3 Raja Narayanan,2 Pradeep Venkatesh,4 Subhadra Jalali2,5 1Vitreoretina and Uveitis Services, L V Prasad Eye Institute, Mithu Tulsi Chanrai Campus, Bhubaneswar, India; 2Srimati Kanuri Santhamma Center for ...
Padhy SK, Takkar B, Narayanan R, Venkatesh P, Jalali S   +4 more
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Shifting Perceptions of CRISPR

Voices in Bioethics, 2021
Photo by National Cancer Institute on Unsplash INTRODUCTION More than 20 cell and gene therapies are now available to safely minimize genetic diseases such as retinal dystrophy (LUXTURNA), some B-cell lymphomas (YESCARTA), and B-cell lymphoblastic ...
Camille Castelyn
doaj   +1 more source