This year’s Nobel Prize in Chemistry was awarded to two female chemists who developed genetic scissors CRISPR, which is considered to the most innovative technology in biotechnology. CRISPR edits DNA, which contains critical information about the organism, by cutting away precisely targeted elements of DNA that cause genetic disorders. As the accuracy and efficiency of the CRISPR technology have increased lately, its potential to treat congenital genetic disorders is improving.
A team led by Professor Krzysztof Palczewski and mainly composed of South Korean researchers at the Gavin Herbert Eye Institute of the University of California, Irvine successfully improved the efficiency of editing the gene that causes Leber congenital Amaurosis, a form of congenital disorder, using the base editor technologies that edit a single base of DNA, according to the international journal Nature Biomedical Engineering’s online edition published on Monday (local time).
Leber congenital Amaurosis is a congenital eye disorder present in infants that is caused by RPE65 gene mutations. Those born with the disorder tend to gradually lose eyesight and become blind in their teens or 20s.
Professor Palczewski’s team succeeded in editing the RPE65 gene in mice with Leber congenital Amaurosis using the base editor technologies at 29-percent efficiency. The existing CRISPR technologies’ success rate for gene editing was only around one to two percent. The optic nerves connecting the retina to the brain of the mice that could not detect light nor distinguish directions and sizes were restored after gene editing.