Results 91 to 100 of about 189,868 (219)
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
Revista Brasileira de Oftalmologia, 2010 A retinosquise juvenil ligada ao cromossomo X é uma degeneração vítreorretiniana hereditária e recessiva. Sua manifestação ocular é a maculopatia cística bilateral e a delaminação das camadas de fibras nervosas da retina.
Luiza Müller Caye +4 more
doaj +1 more source
Макуларна ретиношиза и нејзината семиотска важност [PDF]
, 2015 Вовед. Поимот ретиношиза означува раздвојување во слоевите на неуроретината. Може да се случи во периферните делови на ретината, но исто така и во регионот на макула лутеа кога зборуваме за макуларна ретиношиза.
Blazevska, Karolina +3 more
core
A Synthetic Transcriptional Activator of Genes Associated with the Retina in Human Dermal Fibroblasts. [PDF]
, 2015 Small molecules capable of modulating epigenetic signatures can activate the transcription of tissue-restricted genes in a totally unrelated cell type and have potential use in epigenetic therapy.
Bando, Toshikazu +8 more
core +1 more source
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 +3 more
wiley +1 more source
A Female Case of X-Linked Retinoschisis with Macular Hole Bilaterally
Case Reports in Ophthalmological Medicine, 2020 Purpose. We aimed at reminding that X-linked retinoschisis may also be seen in female patients and share our vitreoretinal surgical experience. Methods. The patient underwent pars plana vitrectomy including the closure of the macular holes with inverted ...
A. Altun
doaj +1 more source
Macular cysts in bietti crystalline dystrophy
TNOA Journal of Ophthalmic Science and Research, 2022 Bietti crystalline dystrophy (BCD) is a rare autosomal disorder characterized by the presence of intraretinal crystalline deposits and later chorioretinal degeneration. We describe a case of BCD with macular cysts.
Syed Mohideen Abdul Khadar +2 more
doaj +1 more source
Decoding the Genetic Puzzle of Inherited Retinal Dystrophies: Novel Insights From a Turkish Cohort
Clinical Genetics, Volume 108, Issue 5, Page 532-552, November 2025.This study analyzes 94 IRD patients from a Turkish cohort using a 141‐gene NGS panel, achieving a 74% diagnostic yield. The identification of 28 novel variants highlights the genetic diversity of IRDs in Türkiye and underscores the value of population‐specific molecular testing.
Şenol Demir +7 more
wiley +1 more source
Advanced Science, Volume 12, Issue 33, September 4, 2025.Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu +7 more
wiley +1 more source
Translational Retinal Research and Therapies. [PDF]
, 2018 The following review summarizes the state of the art in representative aspects of gene therapy/translational medicine and evolves from a symposium held at the School of Veterinary Medicine, University of Pennsylvania on November 16, 2017 honoring Dr ...
Aguirre, Gustavo D +7 more
core +1 more source