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Correcting Genetic Spelling Errors With Next-Generation Crispr
Treatments for rare diseases are hard to create and expensive to deliver, but there is new hope for editing the software of the genome.
Afflicted with the rare disease called progeria, his body aged at a rapid rate, and he died of heart failure at just 17, a brave life cut much too short. My lab discovered the genetic cause of Sam’s illness two decades ago: Just one DNA letter gone awry, a T that should have been a C in a critical gene called lamin A. The opportunity to address this illness by directly fixing the misspelling in the relevant body tissues was just science fiction a few years ago.
Or read this on Wired