Cure

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

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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

Boy with Rare Genetic Disorder Amazes Doctors After World-First Gene Therapy

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Boy with Rare Genetic Disorder Amazes Doctors After World-First Gene Therapy

Courtesy of Oliver Chu family The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental defect called Hunter syndrome. Several years in the making, Oliver Chu became the first in the world to receive the stem cell-based treatment in February, and 3 months later seemed to be … Read more