Sustained improvement in dark-adapted sensitivity but not BCVA after voretigene neparvovec treatment in a mainland Chinese child with biallelic RPE65-associated LCA2: a case report [PDF]
Frontiers in PharmacologyInherited retinal dystrophies (IRDs) represent heterogeneous genetic eye disorders frequently linked to RPE65 mutations. Voretigene neparvovec (VN, Luxturna), an AAV2-mediated gene therapy, remains the sole approved drug for biallelic RPE65-associated ...
Yue Ren +12 more
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Viral and non-viral vectors in gene therapy: current state and clinical perspectives [PDF]
EBioMedicineSummary: Advancements in gene therapy have achieved significant milestones in treating human diseases, offering renewed hope to patients with limited options.
Guannan Geng +6 more
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Mechanisms and Functions of Chromophore Regeneration in the Classical Visual Cycle: Implications for Retinal Disease Pathogenesis and Therapy [PDF]
Biomolecules11-cis-retinal, the indispensable chromophore of photoreceptor opsins, is fundamental for light detection and the initiation of visual signal transduction.
Xinyue Yu +3 more
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Assessment of the official national insurance coverage of regenerative medical products for ophthalmic diseases in Japan following regulatory approval [PDF]
Regenerative TherapyIntroduction: Recently, significant progress has been made in the field of regenerative medicine in Japan for the treatment of ophthalmic diseases, with a notable emphasis placed on clinical research and practical applications, and in 2014, one ...
Kenichi Kimura +3 more
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Gene therapy in retinal diseases: A review
Indian Journal of Ophthalmology, 2021 Over 2 million people worldwide are suffering from gene-related retinal diseases, inherited or acquired, and over 270 genes have been identified which are found to be responsible for these conditions.
Deven Dhurandhar +3 more
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Gene Therapy Medicinal Products: Non-clinical Safety Studies
Безопасность и риск фармакотерапии, 2023 Currently, gene therapy medicinal products (GTMPs) are actively developed in many countries, including the Russian Federation. However, the use of GTMPs raises class-specific safety concerns.The aim of the study was to determine the main requirements for
O. V. Astapova, A. A. Berchatova
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Российский офтальмологический журнал, 2023 Purpose: to evaluate the results of gene therapy by the recombinant adeno-associated viral vector voretigene neparvovec (VN) in children with follow-up periods of 1, 3, 6, and 12 months.Material and methods. The study included 6 children (12 eyes) aged 5
V. V. Neroev +13 more
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Therapeutic landscape for inherited ocular diseases: current and emerging therapies
Singapore Medical Journal, 2023 Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies.
Hwei Wuen Chan, Jaslyn Oh, Bart Leroy
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Validation of a quantitative cell-based relative potency assay for LUXTURNA [PDF]
Molecular Therapy - Methods and Clinical DevelopmentKatherine A. High +2 more
exaly +2 more sources