Results 41 to 50 of about 2,056 (145)
An in vitro enzymatic assay to elucidate the VUS problem in RPE65, a target for retinal gene therapy
, 2023 In 2017 and 2018, Luxturna received FDA and EMA approval as the first gene therapy product to treat patients with biallelic RPE65-mutations, causing severe inherited blindness.
De Baere, Elfride +5 more
core +1 more source
Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
Clinical Ophthalmology, 2020 Jie Gao,1 Rehan M Hussain,2 Christina Y Weng1 1Department of Ophthalmology, Baylor College of Medicine, Houston, TX, USA; 2Retina Associates, Elmhurst, IL, USACorrespondence: Christina Y WengBaylor College of Medicine, Alkek Eye Center, 1977 Butler Blvd,
Gao J, Hussain RM, Weng CY
doaj
Recent Progress in European Advanced Therapy Medicinal Products and Beyond
Frontiers in Bioengineering and Biotechnology, 2018 Cell- and gene-based therapies form one of the pillars of regenerative medicine. They have the potential to transform quality of life and improve the health status of patients with genetic and cellular defects, including genetic diseases ...
Tracy T. L. Yu +6 more
doaj +1 more source
Clinical manifestations of dual‐gene variants in retinitis pigmentosa
Acta Ophthalmologica, EarlyView.Abstract Purpose Retinitis pigmentosa (RP) is an inherited retinal disease (IRD), whereby each affected individual typically harbours pathogenic variants in a single causative gene, yet the disorder exhibits marked genetic heterogeneity, with more than 100 genes reported to underlie RP.
Lasse Wolfram +11 more
wiley +1 more source
Techniques for subretinal injections in animals
Veterinary Ophthalmology, Volume 28, Issue 2, Page 506-518, March 2025.Abstract Subretinal injections are not commonly performed during clinical treatment of animals but are frequently used in laboratory animal models to assess therapeutic efficacy and safety of gene and cell therapy products. Veterinary ophthalmologists are often employed to perform the injections in the laboratory animal setting, due to knowledge of ...
Ryan F. Boyd, Simon M. Petersen‐Jones
wiley +1 more source
An Expanded Toolbox for Versatile Chemical Editing of Adeno‐Associated Virus
Angewandte Chemie, Volume 138, Issue 7, 9 February 2026.We describe technology to introduce diverse non‐natural chemical functionalities site‐specifically into the capsid of adeno‐associated virus through genetic code expansion, and using them to engineer this leading vector for human gene therapy for enhanced tissue specificity and reduced immunogenicity Abstract Site‐specific incorporation of noncanonical
Quan Pham +6 more
wiley +2 more sources
Status and future of recombinant adeno‐associated virus vector manufacturing
Biotechnology Progress, Volume 42, Issue 5, September/October 2026.Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley +1 more source
, 2022 Background: Novartis submitted their health technology assessment in 2019 to the Norwegian Medicine Agency (NOMA), however, in 2020, NOMA discarded the submission by Novartis as they deemed Voretigene Neparvovec (Luxturna) to not be cost-effective ...
Nedberg, Christoffer
core
Compensatory Cross-Modal Plasticity Persists After Sight Restoration
Frontiers in Neuroscience, 2020 Sensory deprivation prompts extensive structural and functional reorganizations of the cortex resulting in the occupation of space for the lost sense by the intact sensory systems. This process, known as cross-modal plasticity, has been widely studied in
Theresa G. Mowad +13 more
doaj +1 more source
Affordability of in vivo gene therapy. Problems and potential solutions
Фармакоэкономика, 2020 Aim. To review foreign approaches to the evaluation and financing of in vivo gene therapy drugs.Materials and Methods. The first stage of the review included the systematic search for publications of clinical-economic studies (CES) on in vivo gene ...
V. V. Omelyanovskiy +4 more
doaj +1 more source