Clinical Genetics Technology, Advanced Diploma, Full-time (5670ADVDIP)

Gene Editing Mechanism of CRISPR-Cas9

When baby KJ was diagnosed with a rare metabolic disorder, our team had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On the new episode of her podcast, Breaking Through, our CEO Madeline Bell speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ’s first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. You can listen to the episode here: http://ms.spr.ly/

Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story