Using new gene-editing technology, David Liu is on his way to treating, and possibly eradicating, thousands of diseases. Adam Zwan is one of just 18 people in the United States battling a rare genetic disease called Wolfram syndrome, which causes childhood diabetes and hearing and vision loss. The average life expectancy for sufferers is 30 to 40 years, and Zwan is 30. Growing up, he took nine different prescription medications but has managed, through diet and exercise, to bring that number down to three. Still, there is no known cure for Wolfram syndrome. Enter David Liu, a chemistry and chemical biology professor at Harvard and senior associate member of the Broad Institute of Harvard and MIT. Liu has developed a new class of DNA base editors that can switch a single letter of DNA with another, opening the door to treatments, and possibly cures, for thousands of genetic diseases caused by one small “typo” in DNA. Think of it as CRISPR 2.0, a new iteration of the powerful gene-editing tool that could alter the genomic structure of mutations associated with diseases like sickle cell anemia, hemophilia, genetic blindness and more. Years of research are needed before any kind of drug therapy could reach the market, but Harvard has granted a worldwide license to Beam Therapeutics Inc., co-founded by Liu, to develop and commercialize a suite of DNA base editing technologies for treating human disease. |