Researchers have developed a groundbreaking “mini gene” that holds promise as a gene therapy for Usher Syndrome type 1F. The new therapy has been tested on mice and has been shown to increase the production of a crucial protein that is responsible for the deafness and progressive vision loss associated with Usher 1F. This research marks a significant step towards the development of a treatment for Usher 1F blindness, which currently has no cure.
Because vision loss in Usher 1F involves a slightly different form of the same protein, the researchers say the same approach may be useful for preventing blindness. These problems arise due to a mutation that interferes with the production of a protein called protocadherin-15, which has slightly different forms in the ear and eye and is needed for cells in the auditory and visual systems to function properly.
So the researchers decided to explore another option: shortening the DNA to create a mini gene that still codes for functional protein yet is small enough to fit inside the viral capsule.The first step involved painstakingly mapping all 25,000 atoms in the external structure of inner-ear protocadherin-15 — a process carried out by co-senior author Marcos Sotomayor, a former research fellow at HMS and now associate professor of chemistry and biochemistry at Ohio State.
Indzhykulian tested the eight mini genes on inner-ear cells in a dish. He confirmed that truncated versions of protocadherin-15 made from mini-gene DNA did bind to cadherin 23, its protein partner in hair cells. “We were all pleasantly surprised,” Corey said. “We thought it would take years of optimizing and trying things and tweaking the protein structure, but this one version pretty much worked.”
Why start by testing the mini gene in the mouse’s inner ear if treating vision loss is the main goal?
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