Areas of Focus

Gene therapies would likely halt further retinal degeneration and possibly restore some vision already lost.

Gene replacement:
Similar to Luxturna, the first FDA-approved gene therapy for an inherited retinal disease, the entire mutated gene is replaced with a good copy. Because the Usher 1F gene is too large to fit on a single viral vector, which is the delivery mechanism, our researchers are developing two different approaches:

• Splitting the gene into two pieces, with the two pieces reassembling once in the eye
• Mini genes, comprised of only those parts of the gene necessary for vision

Gene editing:

• CRISPR-Cas9, which would snip out the mutated portion and insert a corrected piece
• Prime editing, which is similar to erasing the mutation and writing in the correct genetic sequence.

• Retinal cell protection
• Those that will cause the genetic mutation to be ignored.