Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

CRISPR has been used to create a genetic therapy option for a child born in Pennsylvania with a rare metabolic disorder. Unable to convert ammonia to urea, newborn KJ was in serious risk of brain or liver damage, and had to be kept on medications and an extremely restrictive diet to avoid protein metabolism. Children’s […] The post Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’ appea…