Usher syndrome1/17/2024 The work will provide a solid foundation for understanding the function of each usherin isoform and developing an effective gene therapy platform to treat USH2A associated visual defects, she said in the news release. “Understanding which isoforms of usherin are expressed in the retina and the cochlea and what role they play (in contrast to mutant pathogenic forms) is essential in developing an effective gene therapy construct,” Naash said in the release. Naash has already cloned two usherin isoforms to be tested with her innovative platform to safely advance gene therapy for USH2A. To rescue vision loss, Naash’s non-viral therapy targets the mutation in usherin, the protein product that causes Usher Syndrome Type 2A. “Developing an effective treatment for USH2A has been challenging due to its large coding sequence (15.8 kb) that has precluded its delivery using standard approaches and the presence of multiple isoforms with functions that are not fully understood,” said Naash, who will also evaluate the long-term efficacy of the best therapeutic platform for future translation to the clinic, according to the news release. The Usher syndromes (USH) comprise genetically-related disorders that bring about simultaneous hearing loss and vision loss (resulting from an eye condition. Intravitreal treatment consists of injections directly into the vitreal chamber of the eye. Gene therapy is the introduction of a normal gene into cells to correct genetic disorders. “Our goal is to advance our current intravitreal gene therapy platform consisting of DNA nanoparticles/hyaluronic acid nanospheres to deliver large genes in order to develop safe and effective therapies for visual loss in Usher Syndrome Type 2A,” Naash said in the news release. RP affects the retina, the eye’s light-sensitive layer, leading to a breakdown of cells in the retina which causes blindness. Dunn Endowed Professor of biomedical engineering, $1.6 million to support her work.Īccording to a University of Houston news release, Usher Syndrome Type 2A, caused by mutations of the USH2A gene, can include hearing loss from birth and progressive loss of vision, prompting retinitis pigmentosa (RP). The National Eye Institute has awarded Muna Naash, PhD, John S. With the hopes it will restore vision loss in Usher Syndrome Type 2A (USH2A), a rare genetic disease.
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