Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’
Dr. Kiran Musunru (left) and Dr. Rebecca Ahrens-Nicklas (right) led the researchers who developed a personalized treatment for baby KJ – Released CHOP and Penn 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 … Read more