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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.

Novel dual-vector delivery system for Stargardt disease gene therapy

The American biotechnology company Applied Genetic Technologies Corporation (AGTC), specialized in the development of gene therapies for rare diseases, has just announced Stargardt disease as its new preclinical gene therapy program in ophthalmology. Stargardt disease characterized by a progressive loss of central vision affects one in 8,000-10,000 individuals and is the most common form of inherited macular degeneration. The first...

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Researchers devise a novel, high-precision, gene-editing tool to fix genetic diseases

18/11/19 - Gene editing, Gene Therapy,

In recent years, a powerful gene-editing technology called CRISPR-Cas9 has revolutionized biology turning the process of genome modification into an easy and inexpensive process. This revolutionary technique makes use of molecular tools that allow precise identification of DNA fragments –for example a gene– and either introduce modifications, delete a genomic fragment, or even insert a new DNA fragment or a...

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Researchers develop a new non-invasive route to deliver gene therapy to the retina

Researchers at the Johns Hopkins University have tested an alternative route to deliver gene therapy vectors to the retina that could potentially help patients with age-related macular degeneration (AMD) and inherited retinal disorders (IRDs). The new route of administration, called suprachoroidal injection, has been investigated initially in preclinical experiments in animals and uses a microneedle to inject gene-carrying therapeutic viruses...

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Gene therapies open very promising scenarios for the treatment of genetic blindness

Not so far ago, a patient that was clinically diagnosed with a retinal inherited dystrophy had to face an unavoidable outcome, eventual blindness. No effective treatment to cure or halt the progression of the disease was available. This gloomy perspective has been completely upturned by the implementation of cutting edge technologies, and the future looks promising for patients and their...

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New RNA therapy for autosomal dominant retinitis pigmentosa

The United States Food and Drug Administration (FDA) has just approved the designation of the QR-1123 therapy by the Dutch company ProQR as a new investigational drug. This drug aims to treat a form of autosomal dominant retinitis pigmentosa (RPad) caused by the P23H mutation in the rhodopsin gene (RHO). QR-1123 is an antisense oligonucleotide, that is, a short RNA...

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A race against the clock for Leber congenital amaurosis gene therapy

ProQR Sepofarsen therapy On July 29, the Dutch company ProQR announced that the European Medicines Agency (EMA) has granted access to the Agency's priority medicine program (PRIME) to its Sepofarsen therapy (QR-110), designed for the treatment of Leber congenital amaurosis (LCA10), a childhood blinding disorder, caused by the c.2991+1655A>G (p.Cys998 *) mutation in the CEP290 gene. The EMA PRIME program...

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