[HTML][HTML] X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms

RS Molday, U Kellner, BHF Weber - Progress in retinal and eye research, 2012 - Elsevier
X-linked juvenile retinoschisis (XLRS, MIM 312700) is a common early onset macular
degeneration in males characterized by mild to severe loss in visual acuity, splitting of retinal …

Gene therapy in inherited retinal diseases: an update on current state of the art

A Amato, A Arrigo, E Aragona, MP Manitto… - Frontiers in …, 2021 - frontiersin.org
Background: Gene therapy cannot be yet considered a far perspective, but a tangible
therapeutic option in the field of retinal diseases. Although still confined in experimental …

Retinal AAV8-RS1 gene therapy for X-linked retinoschisis: initial findings from a phase I/IIa trial by intravitreal delivery

C Cukras, HE Wiley, BG Jeffrey, HN Sen, A Turriff… - Molecular Therapy, 2018 - cell.com
This study evaluated the safety and tolerability of ocular RS1 adeno-associated virus (AAV8-
RS1) gene augmentation therapy to the retina of participants with X-linked retinoschisis …

The potential of CRISPR/Cas9 gene editing as a treatment strategy for inherited diseases

SA Abdelnour, L **e, AA Hassanin, E Zuo… - Frontiers in cell and …, 2021 - frontiersin.org
Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …

X-linked retinoschisis: an update

SK Sikkink, S Biswas, NRA Parry, PE Stanga… - Journal of medical …, 2007 - jmg.bmj.com
X-linked retinoschisis is the leading cause of macular degeneration in males and leads to
splitting within the inner retinal layers leading to visual deterioration. Many missense and …

[HTML][HTML] Carboxylated nanodiamond-mediated CRISPR-Cas9 delivery of human retinoschisis mutation into human iPSCs and mouse retina

TC Yang, CY Chang, AA Yarmishyn, YS Mao, YP Yang… - Acta biomaterialia, 2020 - Elsevier
Nanodiamonds (NDs) are considered to be relatively safe carbon nanomaterials used for
the transmission of DNA, proteins and drugs. The feasibility of utilizing the NDs to deliver …

Advances in understanding the molecular structure of retinoschisin while questions remain of biological function

JB Heymann, C Vijayasarathy, RN Fariss… - Progress in Retinal and …, 2023 - Elsevier
Retinoschisin (RS1) is a secreted protein that is essential for maintaining integrity of the
retina. Numerous mutations in RS1 cause X-linked retinoschisis (XLRS), a progressive …

Use of dorzolamide for patients with X-linked retinoschisis

MA Apushkin, GA Fishman - Retina, 2006 - journals.lww.com
Purpose: To determine the value of a topical carbonic anhydrase inhibitor for the treatment
of foveal lesions in patients with X-linked retinoschisis (XLRS). Methods: Eight patients with …

Retinoschisin (RS1), the protein encoded by the X-linked retinoschisis gene, is anchored to the surface of retinal photoreceptor and bipolar cells through its …

LL Molday, WWH Wu, RS Molday - Journal of Biological Chemistry, 2007 - ASBMB
Retinoschisin or RS1 is a discoidin domain-containing protein encoded by the gene
responsible for X-linked retinoschisis (XLRS), an early onset macular degeneration …

Of men and mice: human X-linked retinoschisis and fidelity in mouse modeling

C Vijayasarathy, SPBS Pasha, PA Sieving - Progress in Retinal and Eye …, 2022 - Elsevier
X-linked Retinoschisis (XLRS) is an early-onset transretinal dystrophy, often with a
prominent macular component, that affects males and generally spares heterozygous …