How the race to invent a drug for one sick baby made medical history
Briefly

KJ Muldoon's doctors discovered a serious genetic disorder, CPS 1 deficiency, shortly after his birth, putting his life at risk. His parents discussed treatment options, recognizing the severe consequences of this condition. Fortunately, advancements in gene-editing technology, particularly CRISPR, have opened possibilities for personalized medicine targeting rare genetic diseases. A collaborative team at Children's Hospital of Philadelphia and Penn Medicine has been testing new methods to tackle these disorders efficiently, intending to apply this innovative approach to KJ's case, marking an important milestone in the future of genetic treatment.
This was a prime opportunity to do this for real, said Kiran Musunuru, a cardiologist at Penn's Perelman School of Medicine and a co-founder of a gene-editing company, who partnered with Rebecca Ahrens-Nicklas, a pediatrician and director of the Gene Therapy for Inherited Metabolic
Read at The Washington Post
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