When popular YouTube star Adalia Rose died earlier this year, she looked like a diminutive, sickly woman in her 80s. In reality she was only 15 years old, a victim of progeria, an extremely rare genetic disorder caused by a single mutation in one of 3 million base pairs that make up human DNA.
While Rose spent her short life helping to break down the stigma attached to a devastating illness, geneticist David Liu has dedicated his career to developing ways to alter the genetic code that took her life at such a tender age.
“That a single misspelling in her DNA ended Adalia’s life so early is a loss for all of us,” said Liu, a professor of chemistry and chemical biology and director of the Merkin Institute of Transformative Technologies in Healthcare at Harvard University.
…
In his Harvard lab, Liu and his team have invented new ways to repair mutated genes that are less damaging to DNA than prior technologies. One of his lab’s innovations is called a base editor, which he used last year to cure progeria in mice….“The base editor goes into the cells of the animal, looks for the mistake, which in progeria is a C to a T and changes the T back into a C,” Liu told an audience.
