How Gene Editing Is Changing Genetic Disorder Treatment
Via Harvard Magazine
The Story
Harvard chemist David Liu has transformed medicine by inventing gene editing tools that correct genetic mutations without cutting the DNA double helix.
Around 2011 he and graduate student Kevin Esvelt developed PACE, or phage assisted continuous evolution, which harnesses fast reproducing bacteriophages to run 200 generations of protein evolution in about eight days rather than a full year.
Building on that work, his lab created base editing in 2016 with Alexis Komor and later prime editing with Andrew Anzalone, techniques that rewrite individual genetic letters to fix mutations behind diseases like sickle cell anemia.
At least 23 clinical trials now rely on his methods to treat conditions including lung cancer and metabolic disorders.
One patient, a 12 year old English girl named Alyssa Tapley, saw her leukemia driven back in 2022 when her immune cells were edited after conventional treatment failed, and she remains cancer free.
Liu stresses that such breakthroughs depend on sustained funding for curious young researchers, warning that future cures for diseases like progeria hinge on continued investment in basic science.
Around 2011 he and graduate student Kevin Esvelt developed PACE, or phage assisted continuous evolution, which harnesses fast reproducing bacteriophages to run 200 generations of protein evolution in about eight days rather than a full year.
Building on that work, his lab created base editing in 2016 with Alexis Komor and later prime editing with Andrew Anzalone, techniques that rewrite individual genetic letters to fix mutations behind diseases like sickle cell anemia.
At least 23 clinical trials now rely on his methods to treat conditions including lung cancer and metabolic disorders.
One patient, a 12 year old English girl named Alyssa Tapley, saw her leukemia driven back in 2022 when her immune cells were edited after conventional treatment failed, and she remains cancer free.
Liu stresses that such breakthroughs depend on sustained funding for curious young researchers, warning that future cures for diseases like progeria hinge on continued investment in basic science.
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