Save Sight Now Europe has awarded its first grant to the University of Wisconsin-Madison to fund research on gene editing in retinal degeneration. The project, led by Dr. David Gamm, will use CRISPR/Cas9 technology to insert a gene construct into human embryonic stem cells (hESCs) that will allow researchers to study the function of the MYO7A gene in photoreceptors.
MYO7A is a gene that is essential for normal vision. Mutations in the MYO7A gene can cause Usher syndrome, a group of genetic disorders that lead to hearing loss and vision loss. The goal of this project is to use gene editing to create a model of Usher syndrome in hESCs. This model will allow researchers to study how MYO7A functions in photoreceptors and to develop new therapies for Usher syndrome.
“This project is a critical step towards developing new treatments for Usher syndrome,” said Dr. Gamm. “We are excited to be working with Save Sight Now Europe to make this research possible.”
The support from Save Sight Now Europe will fund the first 6 months of this project. If the project is successful, researchers hope to continue their work and eventually develop therapies for Usher syndrome.