Results 51 to 60 of about 1,367 (120)
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
Clinical &Experimental Ophthalmology, Volume 54, Issue 7, Page 917-931, September/October 2026.ABSTRACT Background The lack of validated and sensitive clinical endpoints remains a major challenge in the design of gene therapy trials for inherited retinal dystrophies (IRDs). This prospective longitudinal cohort study describes the natural disease progression of IRDs caused by pathogenic mutations in the Crumbs homologue 1 (CRB1) gene, and ...
Jessica S. Karuntu +15 more
wiley +1 more source
Ciliary Defects in Inherited Retinal Diseases
Advanced Genetics, Volume 7, Issue 1, March 2026.The photoreceptor cilium is a specialized sensory organelle essential for vision. This review systematically summarizes the structural and functional defects of the cilium that lead to inherited retinal diseases (IRDs). It highlights key pathogenic genes, elucidates molecular mechanisms of degeneration, and evaluates emerging therapeutic strategies ...
Guizhi Guo, Lin Li, Jun Zhou, Jie Ran
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Acta Ophthalmologica, Volume 104, Issue 1, Page 89-97, February 2026.Abstract Purpose To evaluate test–retest variability (TRV) of the full‐field stimulus test (FST) in patients with retinitis pigmentosa (RP) and poor best‐corrected visual acuity (BCVA; ≤20/50 Snellen; ≥0.40 logMAR), and to assess the reliability of FST as a clinical endpoint in future RP trials.
J. S. Karuntu +2 more
wiley +1 more source
Human Mutation, Volume 2026, Issue 1, 2026.Osteogenesis imperfecta (OI) is a genetically and clinically heterogeneous bone disorder, with more than 20 genes contributing to OI development. Previously, we identified WNT1 c.620G > A (p.Arg207His) mutation among Chinese patients with autosomal recessive OI (AR‐OI).
Shan Li +10 more
wiley +1 more source
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 +4 more
doaj
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
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Toward Precision Medicine: Gene Therapy Applications in the Management of Uveal Melanoma
Cancer Reports, Volume 8, Issue 12, December 2025.ABSTRACT Background Uveal melanoma (UM) is the prevailing malignant tumor that develops within the eye in adults, and it has a bleak outlook because of the few treatment choices available and the high likelihood of returning after treatment. Currently, surgical intervention, radiation therapy, and a combination of both modalities are available ...
Alireza Azani +13 more
wiley +1 more source
Clinical Pharmacology &Therapeutics, Volume 118, Issue 6, Page 1405-1421, December 2025.Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami +2 more
wiley +1 more source
Clinical and Translational Science, Volume 18, Issue 12, December 2025.ABSTRACT Natural AAV serotypes often lack specificity and efficiency, leading to off‐target effects and a low therapeutic index. To overcome these limitations of naturally occurring serotypes, there has been a keen interest in the field to engineer novel capsids to enhance tissue and cell‐specific targeting, resulting in a high number of published ...
Chinaza Agbim +5 more
wiley +1 more source