Something Positive: Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’ | A Positive Story

Something Positive: Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’ | A Positive Story

Why this story matters: Amid constant bad news, it’s important to highlight examples of progress that rarely make the front page. Quick summary: This story highlights recent developments related to infants, showing how constructive action can lead to meaningful results. 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 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 Hospital of Philadelphia (CHOP) doctors believed they could use CRISPR to develop a treatment to correct a faulty gene in KJ’s genome that would essentially cure him. Would you like to read more good news about Infants , Disease , Genetics , Hospitals , Phi…

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