Results 61 to 70 of about 1,367 (120)

Cell and Gene Therapy: Transforming Treatment Paradigms for Patient‐Centric Care

Clinical and Translational Science, Volume 18, Issue 12, December 2025.
ABSTRACT Cell and gene therapies (CGTs) are transforming medicine by offering potential cures for diseases previously considered untreatable. Despite rapid advancements, challenges remain in optimizing efficacy and safety and ensuring patient accessibility and preference due to high costs and clinical uncertainties, particularly for rare diseases and ...
Sojeong Yi, Chia‐Yung Wu, Avery McIntosh, Maria E. Santaella, Tara M. Robinson, Michael Z. Liao   +5 more
wiley   +1 more source

Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates

Molecular Therapy: Methods & Clinical Development, 2019
We have demonstrated safe and effective subretinal readministration of recombinant adeno-associated virus serotype (rAAV) to the contralateral eye in large animals and humans even in the setting of preexisting neutralizing antibodies (NAbs ...
Lindsey Weed, Michael J. Ammar, Shangzhen Zhou, Zhangyong Wei, Leona W. Serrano, Junwei Sun, Vivian Lee, Albert M. Maguire, Jean Bennett, Tomas S. Aleman   +9 more
doaj   +1 more source

New insights into applications of base editor in hereditary disorders

Interdisciplinary Medicine, Volume 3, Issue 6, November 2025.
Abstract Hereditary disorders are a group of diseases caused by genetic mutations or chromosomal variations. Although the incidence of each genetic disorder is relatively low, patients affected by the disease generally experience a range of severe symptoms, including blindness, disability, and even premature death. In addition, the available treatments
Maoping Cai, Linhui Zhang, Liqing Li, Yue Liu, Canbin Lv, Yan Shi, Jianyuan Zhao, Dingwei Ye, Yuanyuan Qu   +8 more
wiley   +1 more source

Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events—A Systematic Review

Clinical &Experimental Ophthalmology, Volume 53, Issue 8, Page 967-985, November 2025.
ABSTRACT Background To explore the prevalence of serious adverse events (SAEs) associated with retinal viral gene therapy and to examine trends influencing SAE occurrences in human gene therapy surgeries and pre‐clinical animal trials. Methods Literature review was performed to identify peer‐reviewed human and animal studies relevant to viral gene ...
Aubrey Berger, Ciera D. Johnson, Alan D. Marmorstein, Brittni A. Scruggs   +3 more
wiley   +1 more source

Mechanisms and Functions of Chromophore Regeneration in the Classical Visual Cycle: Implications for Retinal Disease Pathogenesis and Therapy

Biomolecules
11-cis-retinal, the indispensable chromophore of photoreceptor opsins, is fundamental for light detection and the initiation of visual signal transduction.
Xinyue Yu, Hao Fan, Hui Zhang, Xiaorong Li   +3 more
doaj   +1 more source

Proposal for a Germline Expert Panel to Improve Variant Reclassification in Japan

Cancer Science, Volume 116, Issue 10, Page 2907-2909, October 2025.
This letter argues that Japan's dependence on a single foreign company for classifying germline variants is delaying critical treatment for cancer patients. The author proposes establishing a national “Germline Expert Panel” to provide independent, timely oversight, and ensure patients receive appropriate care.
Kazuki Yamazawa
wiley   +1 more source

Gene Therapy for Inherited Retinal Disease: Long-Term Durability of Effect

, 2022
International audienceThe recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65 mutation-associated inherited retinal dystrophy with viable retinal cells represents an important step in the development of ocular gene ...
Spera, Claudio, John G. Flannery, Hendrik P.N. Scholl, Claudio Spera, Maclaren, Robert, Dalkara, Deniz, Scholl, Hendrik P.N., M. Dominik Fischer, Viriato, Daniel, Fischer, M. Dominik, Banhazi, Judit, Deniz Dalkara, Flannery, John, Judit Banhazi, Robert E. MacLaren, Bart P. Leroy, Daniel Viriato, Daniel C. Chung, Leroy, Bart, Chung, Daniel   +19 more
core   +1 more source

Retinitis Pigmentosa: From Genetic Insights to Innovative Therapeutic Approaches—A Literature Review

Medicina
Retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal dystrophies characterized by progressive photoreceptor degeneration and vision loss.
Ricardo A. Murati Calderón, Andres Emanuelli, Natalio Izquierdo   +2 more
doaj   +1 more source

A Practical Guide to Genetic Eye Conditions for Paediatricians

Journal of Paediatrics and Child Health, Volume 61, Issue 10, Page 1538-1548, October 2025.
ABSTRACT Introduction Inherited eye disorders, though individually rare, are a collectively common cause of paediatric vision impairment. Many occur as part of a syndrome, in association with congenital anomalies and/or growth/developmental disorders.
Richard Lin, Alan Ma, Benjamin M. Nash, Zachary McPherson, John R. Grigg, Robyn V. Jamieson   +5 more
wiley   +1 more source

Cyclic nucleotide signaling as a drug target in retinitis pigmentosa

FEBS Letters, Volume 599, Issue 18, Page 2557-2570, September 2025.
Disruptions in cGMP and cAMP signaling can contribute to retinal dysfunction and photoreceptor loss in retinitis pigmentosa. This perspective examines the mechanisms and evaluates emerging evidence on targeting these pathways as a potential therapeutic strategy to slow or prevent retinal degeneration.
Katri Vainionpää, Ahmed B. Montaser, Henri Leinonen   +2 more
wiley   +1 more source