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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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 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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World Sight Day is observed annually on the second Thursday of October. This global event coordinated by The International Agency for the Promotion of Blindness (IAPB) in cooperation with the World Health Organization (WHO) is meant to raise public awareness of blindness and vision impairment as major international public health issues. World Sight Day aims to influence governments/ministers of health...
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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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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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