{"id":43632,"date":"2025-01-09T12:40:21","date_gmt":"2025-01-09T11:40:21","guid":{"rendered":"https:\/\/semmelweis.hu\/english\/?p=43632"},"modified":"2025-01-09T12:41:28","modified_gmt":"2025-01-09T11:41:28","slug":"semmelweis-university-involved-in-testing-breakthrough-technology-to-prevent-vision-loss","status":"publish","type":"post","link":"https:\/\/semmelweis.hu\/english\/2025\/01\/semmelweis-university-involved-in-testing-breakthrough-technology-to-prevent-vision-loss\/","title":{"rendered":"Semmelweis University involved in testing breakthrough technology to prevent vision loss"},"content":{"rendered":"
Several researchers at Semmelweis University participated in the study led by Dr. Bence Gy\u00f6rgy and Dr. Botond Roska, which aims to prevent vision loss in patients suffering from a form of inherited macular degeneration. The newly developed gene-editing technique could lead to breakthroughs not only in the treatment of Stargardt disease but also in the therapy of other hereditary retinal diseases. Semmelweis University has been collaborating with world-renowned Semmelweis alumnus Dr. Botond Roska and his institute, the Institute of Molecular and Clinical Ophthalmology Basel (IOB) for more than ten years, whose mission is to cure vision loss and restore eyesight.<\/div>\n

A highly effective gene-editing technique developed by researchers at the Institute of Molecular and Clinical Ophthalmology Basel (IOB) could potentially treat Stargardt disease, the most common form of inherited macular degeneration, which can lead to vision loss. In a paper published in the journal Nature Medicine<\/em> on January 8<\/a>, the researchers detail the successful application of the base-editing technique, which can correct the most common mutation associated with Stargardt disease.<\/p>\n

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Among the authors of the study there are several researchers at Semmelweis University; an important part of the experiments was carried out using a special retinal tissue culture from the Retina Laboratory of the Department of Anatomy, Histology and Embryology. The effectiveness of the base-editing technique \u00a0was demonstrated in human retinal cultures and human retinal pigment epithelial cells, among others. The procedure used in the experiments, which is unique worldwide and enables the survival of retinal tissue harvested from deceased organ donors for up to 35 weeks, had been developed at Semmelweis University by Head of Laboratory Dr. Arnold Szab\u00f3.<\/p>\n

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Image credit: RTL Praxis<\/figcaption><\/figure>\n

The research was led by Dr. Bence Gy\u00f6rgy of IOB, who, like Dr. Botond Roska, graduated from Semmelweis University. He has been collaborating with the Department of Ophthalmology since his undergraduate years, and he started his research career at Semmelweis\u2019s Department of Genetics, Cell and Immunobiology. As he told our website, base editing, this precision gene-editing technique, involves injecting the base-editing enzyme into the retina using the so-called adeno-associated virus (AAV), a non-pathogenic virus vector system, like a Trojan horse. This \u201cmolecular machine\u201d can detect which of the 3 billion base pairs in DNA is defective and correct it. \u201cThis impressive precision is essential for the development of a safe and effective therapy,\u201d said Dr. Bence Gy\u00f6rgy, Head of the IOB’s Ophthalmic Translational Research Group, who is hopeful that the technology could be applied to patients within a few years.<\/p>\n

The researchers tested the technique in seven different models, from cell cultures to human retinal cells. Work of this scale requires several collaborating partners, including Semmelweis University, where the human retina model was tested. \u00a0\u00a0<\/p>\n

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Dr. Arnold Szab\u00f3 (in the middle) with his colleagues<\/figcaption><\/figure>\n

\u201cIn probing the newly published base-editing technique in human models, i.e. human retinal cultures artificially fixed in the laboratory, we have tested the IOB-developed viral vectors in several combinations to measure their effectiveness. The fact that the tests were done in real human tissues provides strong evidence for the future applicability of the treatment in patients,\u201d stressed Dr. Arnold Szab\u00f3, Head of the Retina Laboratory at the Department of Anatomy, Histology and Embryology at Semmelweis University. He added that D\u00e1niel Magda and Ferenc Kilin, who also co-authored the study, had carried out much of the experimental work.<\/p>\n

\"\"<\/a>Dr. Botond Roska highlighted that they had been cooperating with Semmelweis University for more than ten years, especially with Dr. Zolt\u00e1n Zsolt Nagy, Director of the Department of Ophthalmology, and Dr. Arnold Szab\u00f3. The research that has led to the publication is also in line with the project by Dr. Botond Roska and the above-mentioned two researchers from Semmelweis University, which has been granted funding in the framework of the National Research, Development and Innovation Office’s Frontline – Research Excellence Programme launched in 2021<\/a>.<\/p>\n

\"\"<\/a>A gene-editing technique similar to the one just published has not yet been used in patients, but a gene therapy intervention using the AAV vector system is already available for another rare eye condition. Ten patients have already been treated at the Department of Ophthalmology of Semmelweis University, which has been a certified ophthalmic gene therapy center<\/a> since 2022. The results of the AAV vector-based gene therapy are favorable, pointed out Director Dr. Zolt\u00e1n Zsolt Nagy, who is also one of the authors of the current study. Hopefully, the new therapeutic method will soon be available to patients, so that more ophthalmic genetic defects can be cured, he added.<\/p>\n

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As the researchers noted in a press release issued by IOB<\/a>, their base-editing approach could potentially be adapted to treat other inherited retinal diseases caused by similar types of mutations. These results represent a significant advancement in the field of ocular gene therapy.<\/p>\n<\/div>\n\r\n [slb_exclude]\r\n

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The next steps include further safety studies and preparations for clinical trials.\u00a0<\/p>\n

Stargardt disease affects 1 in 6,500 individuals and is the most common form of inherited macular degeneration. It is a hereditary disease, whose symptoms develop at a young age, causing a gradual loss of visual acuity and, eventually, in the majority of cases, legal blindness.<\/p>\n

P\u00e1lma Dobozi
\nTranslation: Judit Szabados-D\u0151tsch
\nPhotos by Bogl\u00e1rka Zellei, Attila Kov\u00e1cs \u2013 Semmelweis University; G\u00e1bor Ancsin, RTL Praxis<\/p>\n","protected":false},"excerpt":{"rendered":"

The new gene-editing technique was developed in a research project led by Dr. Bence Gy\u00f6rgy and Dr. Botond Roska.<\/p>\n","protected":false},"author":102255,"featured_media":43630,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[13,286],"tags":[116,78,210],"class_list":["post-43632","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science","category-x-nyitolap","tag-innovation","tag-international-relations","tag-research-development"],"acf":[],"_links":{"self":[{"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/posts\/43632","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/users\/102255"}],"replies":[{"embeddable":true,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/comments?post=43632"}],"version-history":[{"count":1,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/posts\/43632\/revisions"}],"predecessor-version":[{"id":43633,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/posts\/43632\/revisions\/43633"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/media\/43630"}],"wp:attachment":[{"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/media?parent=43632"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/categories?post=43632"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/semmelweis.hu\/english\/wp-json\/wp\/v2\/tags?post=43632"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}