Expanding gene therapy for Usher Syndrome Type 2A

by admin
0 comment

Researchers at the University of Houston are expanding methods of gene therapy

Muna Naash, PhD, John S. Dunn Endowed Professor of Biomedical Engineering, University of Houston, is expanding gene therapy approaches in hopes of reversing vision loss in Usher syndrome type 2A. (Image courtesy of University of Houston)

In hopes of reversing the vision loss of Usher Syndrome Type 2A (USH2A), a rare genetic disease. awarded $1.6 million to help.

Usher syndrome type 2A, caused by mutations in the USH2A gene, includes birth deafness and progressive visual impairment, and can lead to retinitis pigmentosa (RP), according to a University of Houston news release. RP affects the light-sensitive layer of the eye, the retina, causing destruction of the cells in the retina and causing blindness. There is currently no cure for USH2A.

“Our goal is to evolve the current intravitreal gene therapy platform composed of DNA nanoparticles/hyaluronic acid nanospheres to develop a safe and effective treatment for vision loss in Usher syndrome type 2A. It’s about delivering big genes,” Naash said in a news release.

Gene therapy is the introduction of normal genes into cells to correct inherited diseases. Intravitreal therapy consists of direct injection into the vitreous cavity of the eye.

“Developing effective therapeutics for USH2A is challenging because of its large coding sequence (15.8 kb), which precludes delivery using standard approaches and the development of multiple isoforms with incompletely understood functions. It was difficult because of existence,” said Naash. Long-term efficacy of the best therapeutic platform for future clinical translation, according to a news release.

To rescue vision loss, Naash’s nonviral therapy targets mutations in ucherin, the protein product that causes Usher syndrome type 2A. Naash has already cloned two of her Usherin isoforms that will be tested in her innovative platform to safely advance USH2A gene therapy.

“Understanding which isoforms of usherin are expressed in the retina and cochlea and what role they play (as opposed to mutant pathogens) will help develop effective gene therapy constructs. It’s essential on top of that,” Naash said in a release.

This study will provide a solid foundation for understanding the function of each usherin isoform and for developing an effective gene therapy platform to treat USH2A-associated visual disorders.

You may also like

Leave a Comment