Skip to main content

New Publication from Dr. Matt Hirsch’s Team Strengthens UNC’s MPS I Corneal Gene Therapy Approach

By

A new paper from Dr. Matt Hirsch’s lab shares promising results from a study in cats with MPS VI, a rare disease that causes the cornea to become cloudy and affects vision.

Two cats eyes - one clear that was treated with gene therapy vector (AAV8-optARSB) and the other clouded that was the control.
The treated eye on the left clears after a low-dose intracorneal AAV8-optARSB gene therapy injection, while the untreated eye on the right remains cloudy. Clarity returns within 2–3 weeks and is sustained; when the second eye is treated later, it shows the same recovery.

In the study, the team used a small dose of a gene therapy vector (AAV8-optARSB) injected directly into the cornea. The cloudy corneas began to clear within 2–3 weeks and stayed clear for the rest of the study. When the second eye was treated later, it showed the same improvement.

The treatment method used here is the same one UNC is developing to improve people with MPS I, another rare condition that can cause similar eye problems. Because the study showed strong and lasting improvements in the animals, it offers encouraging evidence that this approach could also be safe and effective for future use in humans.

Read the publication:
https://pmc.ncbi.nlm.nih.gov/articles/PMC12680226/ 
DOI: 10.1371/journal.pone.0338370

Filed Under:

Categories: Uncategorized