Baby treated with first-ever personalized gene editing treatment

A nine-month-old baby who was born with a rare genetic disorder is the first person to be successfully treated with personalized CRISPR gene editing therapy, scientists in Philadelphia announced on Thursday.The big picture: This "historic medical breakthrough" could "provide a pathway for gene editing technology to be successfully adapted to treat individuals with rare diseases for whom no medical treatments are available," per statement from the University of Pennsylvania and the Children's Hospital of Philadelphia.While KJ "is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs," said a Rebecca Ahrens-Nicklas, an assistant professor of pediatrics and genetics at the Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania who treated the infant, in a statement.Driving the news: KJ was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, per according from the statement from the hospital and the university's Penn Medicine.What they did: Researchers "corrected a specific gene mutation in the baby's liver cells that led to the disorder," per a Thursday statement from the National Institutes of Health (NIH), which supported the research, the findings of which were published in The New England Journal of Medicine on Thursday. After spending the first several months of his life in the hospital, KJ received the first dose of his bespoke therapy in February.He is "now growing well and thriving" after the treatment was safely administered, per the hospital and the university's Penn Medicine.Context: The research that scientists are hailing as "historic" comes after years of progress in gene editing and decades of federally funded research.Gene editing based on CRISPR, which stands for clustered regularly interspaced short palindromic repeat, can precisely correct disease-causing variants in the human genome."CRISPR is an advanced gene editing technology that enables precise changes to DNA inside living cells," according to the NIH."This is the first known case of a personalized CRISPR-based medicine administered to a single patient and was carefully designed to target non-reproductive cells so changes would only affect the patient."What they're saying: "As a platform, gene editing — built on reusable components and rapid customization — promises a new era of precision medicine for hundreds of rare diseases," said Joni Rutter, director of NIH's National Center for Advancing Translational Sciences, in a statement.It's "bringing life-changing therapies to patients when timing matters most: Early, fast, and tailored to the individual," Rutter added.The bottom line: "This truly is the future for all of these gene and cell therapies," said Arkasubhra Ghosh, who studies gene therapy at Narayana Nethralaya Eye Hospital in Bengaluru, India, per Nature. "It's really exciting," added Ghosh, who wasn't involved in the study.Go deeper: Gene therapy's slow rollout offers a reality check

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