A Baby Benefits From Personalized Gene Editing in the Clinic

A baby benefits from personalized gene editing in the clinic

An infant with an inherited life-threatening metabolic condition has responded to a tailored therapy that uses the gene-editing tool CRISPR to correct a specific mutation. An infant with an inherited life-threatening metabolic condition has responded to a tailored therapy that uses the gene-editing tool CRISPR to correct a specific mutation.

Gene-Therapy Treatment Of Infant With Rare Genetic Condition Successful In World First

Pexels When baby KJ was born with a rare disease, which could cause a potentially fatal amount of ammonia to build up in his body, damaging his organs in the process, his parents were told he would require a liver transplant when he was old enough, and would need a restricted diet and frequent hospital monitoring in the meantime. Then, they were approached by medical researchers at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine…