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"R-Tech Ueno Ltd. (Tokyo, Japan) announced it has completed a phase 2 clinical trial of 0.15% UF-021 isopropyl unoprostone (Ocuseva), which is under development as a treatment for retinitis pigmentosa (RP). The trial investigated the possibility of improving visual function in the central part of the retina with UF-021 in patients with RP."
"The retinal sensitivity measured with the Humphrey perimeter showed statistically significant improvement at weeks 4 and 8 in the two-drops-per-time group compared with the placebo group."

Francois Paquet-Durand, PhD, chief scientific officer at the company Mireca, discusses an emerging drug for retinitis pigmentosa, and other inherited retinal diseases.

"A discovery opens the possibility of one day restoring loss of vision by activating the retina’s ability to regenerate. Researchers at Baylor College of Medicine, the Cardiovascular Research Institute and the Texas Heart Institute reveal in the journal Cell Reports that although the mammalian retina – a layer of specialized nerve cells that mediates vision and is located on the back of the eye– does not spontaneously regenerate, it has a regenerative capacity that is kept dormant by a cellular mechanism called the Hippo pathway. The discovery opens the possibility of activating the retina’s ability to restore lost vision by manipulating this pathway."

"The aim of this study is to analyze the effect of nutraceutical molecules with antioxidant properties, on the progression of the disease in an established animal model of RP, and rd10 mice. We show that chronic treatment per os with a flavanone (naringenin) or a flavonol (quercetin) present in citrus fruits, grapes and apples, preserves retinal morphology, and ameliorates functionality. These actions are associated with a significant reduction of stress-oxidative markers, such as the detoxifying enzymes Sod1 and Sod2. In addition, naringenin and quercetin treatment reduces the levels of acrolein staining associated with a reduction of ROS in the cellular environment. The study demonstrates the beneficial effects of naringenin and quercetin, two molecules that possess antioxidant properties, limiting neurodegeneration, and thus preventing cone damage."

"It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems. They were able to see motion, brightness changes over a thousandfold range and fine detail on an iPad sufficient to distinguish letters."

Toward a therapy for deafness and blindness in Usher syndrome

Two HMS neurobiologists studying the origins of deafness—Corey and Artur Indzhykulian, HMS assistant professor of otolaryngology at Massachusetts Eye and Ear—are joining forces with Botond Roska, an expert on retinal biology and eye disease at the Institute of Molecular and Clinical Ophthalmology in Basel, Switzerland, to develop treatments for a form of Usher syndrome.

Corey and Indzhykulian’s work will focus on a particularly severe form of the disease, known as Usher syndrome type IF.

Eloxx Pharmaceuticals is developing small molecules that permit read-through of point mutations that cause Usher syndrome 1F and 2A.

from Globe Newswire. The AP news staff was not involved in its creation.
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Gene therapy
Globe Newswire
GlobeNewswire: Medical
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Eloxx Pharmaceuticals Presents Positive New Data at the Association for Research in Vision and Ophthalmology “ARVO” 2019 Annual Meeting
Eloxx Pharmaceuticals today. Eloxx is evaluating several of its eukaryotic ribosomal selective glycoside (ERSG) molecules in IND-enabling studies for use in the treatment of inherited retinal diseases with an initial focus on Usher Syndrome

Preclinical studies have demonstrated:dose-dependent restoration of missing protein of Usher Syndrome nonsense mutations;encouraging pharmacokinetics in the retina by intravitreal injection;a favorable tolerability profile at high doses in sensitive species

2Ctech, Inc., a privately-held development stage company focused on the application of nanoparticle technologies for the treatment of retinal diseases, announced the initiation of the first-known clinical program to demonstrate the effectiveness and safety of Quantum Dots (QDs) to achieve photovoltaic stimulation of the neural retina for preservation or enhancement of vision in patients with retinal degenerative diseases and, in particular, Retinitis Pigmentosa (RP). In previous pre-clinical and first-in-human trials, a one-time injection of QDs was administered into the vitreous and trial outcomes provided strong evidence of safety and positive indications of meaningful vision enhancement. The next human studies are intended to meet the intent of FDA’s Early Feasibility Study (EFS) program requirements and will be conducted at several US sites.

Scientists describe a method of rendering the gene editing tool CRISPR-Cas9 'immunosilent,' potentially allowing the editing and repair of genes to be accomplished reliably and stealthily.