Will editing genetic typos cure blindness?

A new gene-editing technique holds the potential to revolutionize how inheritable diseases are treated by allowing physicians to correct mutations quickly. CRISPR, or “clustered regularly interspaced short palindromic repeats,” adapts a bacterial defense mechanism of identifying and disabling DNA and uses it to fix genetic “typos.”

Stephen Tsang, the László Z. Bitó Associate Professor of Ophthalmology, Pathology, and Cell Biology at Columbia University Medical Center, along with other ophthalmologists from Columbia and the University of Iowa, used this technique to repair a genetic flaw that causes a condition that produces tunnel vision or complete blindness. 

The researchers have already successfully modified stem cells from the skin of a person suffering from the condition. If the FDA approves the procedure, investigators would inject the corrected stem cells into a patient’s retina. Their hypothesis is that the stem cells will grow into healthy tissue, restoring the person’s vision. Learn More.