Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality ...

World’s first: Gene editing clinically deployed for breakthrough treatment of rare genetic disease led by @kiranmusunuru & Rebecca Ahrens-Nicklas www.nejm.org/doi/full/10.... #GeneEditing #CRIPR #OrphanDisease #CPS1deficiency #TranslationalScience