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Noticias y Eventos

Descubra las novedades en el campo de las patologías oculares hereditarias

Blog operado por el equipo de DBGen que actualiza terapias, noticias y eventos relacionados con las enfermedades hereditarias oculares. Le invitamos a participar en este foro.

Optogenetic therapy to restore advanced vision loss in retinal dystrophies

Gene therapies currently under study for retinal dystrophies are administered when viable photoreceptor able to be rescued at the functional level still remain. Most therapies are gene-specific and, in some cases, mutation-specific. In advanced disease stages, when a large number of photoreceptor cells have degenerated, the probability of success of these therapies is very low. Therefore, researchers are exploring methodologies...

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Encouraging results from achromatopsia gene therapy trials

Achromatopsia is a disorder that affects one in every 30,000 people and is characterized by the partial or total absence of colour vision due to lack of function of cones, one type of retinal cells. Achromatopsia patients have other visual problems such as extreme sensitivity to light (photophobia), uncontrollable and involuntary eye movements (nystagmus) and decreased visual acuity. Achromatopsia is...

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Progress in anti-VEGF gene therapy for age-related macular degeneration

Age-related macular degeneration (AMD) is one of the most common disease of the retina and the main cause of vision loss in adults over 60 years. It is estimated that 3 million people worldwide are affected, and its incidence increases as life expectancy grows in developed countries. AMD is a progressive disease that affects the macula, the area near the...

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Advances in gene therapy for retinitis pigmentosa due to RPGR gene mutation

In the first months of 2020, positive results in gene therapy of X-linked retinitis pigmentosa (XLRP) have been reported. XLRP is an inherited genetic disorder that causes progressive vision loss, mainly in men. It begins in childhood with night blindness, progresses with the gradual reduction of the visual field and visual acuity, and leads to blindness in the fourth decade...

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A revolutionary CRISPR gene editing technique applied for the first time to a patient with a visual disorder

The patient, treated at the Casey Eye Institute in Portland, Oregon, belongs to a group of 18 people affected with Leber congenital amaurosis. These patients will participate in the BRILLIANCE study, the first clinical trial to determine the safety and efficacy of a therapy based on the CRISPR-Cas9 gene editing technique (AGN-151587, EDIT-101). It is the first example of an...

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First gene therapy application of Luxturna in the UK

This February Moorfields Eye Hospital in London and Radcliffe Hospital in Oxford have started treating patients affected by Leber congenital amaurosis (LCA) with the Luxturna gene therapy. LCA is a childhood-onset severe retinal dystrophy, which causes irreversible blindness for which, until now, no treatment was available. Luxturna (voretigene neparvovec) is also the first gene therapy approved for the treatment of an...

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