Inherited Retinal Diseases
Stargardt disease, retinitis pigmentosa, and other inherited retinal diseases can affect people at any age, with symptoms typically worsening over time. These conditions are caused by mutations that disrupt the activity of specific genes.
For example, people with Stargardt disease carry a mutation in a single gene that leads to the death of cells in the retina. IOB researchers have developed a groundbreaking gene therapy using molecular tools called base editors to correct faulty DNA and fix the most common genetic mutation causing Stargardt disease. IOB scientists have successfully delivered these base editors to human retinal cells and corrected the mutations, both in human retinas and in miniaturized retinas, called organoids, that are grown from Stargardt patients in the laboratory.
IOB researchers have developed a gene therapy that uses molecular machines called base editors to change faulty DNA snippets and revert the most common genetic mutation that causes Stargardt disease back to normal.
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