Dr. Jordi Monés, MD, PhD, the Medical Director of the Barcelona Macula Foundation and an ophthalmologist who specialises in macula, retina and vitreous, gave a masterclass on developments in stem cells and gene therapy to fight blindness on 14 November at the 14th Congress of the European School for Advanced Studies in Ophthalmology (ESASO). The event in Istanbul drew a thousand of the top specialist ophthalmologists from fifty countries.
As Dr. Monés explained, the research being carried out on animal experimental models –and which is beginning to move towards the first clinical trials on humans in countries like the United States, the United Kingdom, France and Japan– includes regenerative therapy with stem cells from many separate sources, not only embryonic but also from the patient him/herself, from donors and from post-mortem eye banks. In addition, gene therapy (genetic manipulation) has corrected some defects in diseases that cause blindness. Progress has also been made in combining cellular regenerative therapy and gene therapy, where stem cells, having been obtained from the patient, are manipulated genetically to correct their defects and subsequently reintroduced. However, while all these techniques lead to great optimism, they are not free of risks and major adverse effects such as the genesis of tumours, genetic mutations and inmunocompatibility problems.
There is another very ambitious line of research, one that could overcome some of the aforementioned limitations and difficulties: optogenetics. Accordingly, the Barcelona Macula Foundation: Research for Vision, with the full involvement and participation of other research centres in Spain, is leading, coordinating and developing a research project to fight unpreventable blindness with the aim of giving blind patients their sight back.
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