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MedEdge MEA > Video > Revolutionizing Pediatric Blood Disorders: Gene Therapy Insights | Lara Khouri: EPHOC
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Revolutionizing Pediatric Blood Disorders: Gene Therapy Insights | Lara Khouri: EPHOC

ME Desk
ME Desk
Published: September 1, 2025
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Lara Khouri, President and Chief Operating Officer of Childrenโ€™s Hospital Los Angeles (CHLA), to discuss the hospitalโ€™s mission and groundbreaking innovations in pediatric care.

Lara highlighted that CHLA has been dedicated to caring for children and youth for nearly 125 years as a leading academic medical center that integrates clinical care, research, and education. Ranked among the top childrenโ€™s hospitals in the United States and globally, CHLA provides family-centered care and treats over 6,200 patients each year through its renowned Cancer and Blood Disease Institute.

A major focus of the discussion was gene therapy, which Lara described as a โ€œrevolutionary step forwardโ€ in treating genetic blood disorders like sickle cell disease and thalassemia. CHLA is one of the few centers in the U.S. capable of offering these cutting-edge therapies, where patient stem cells are collected, genetically corrected, and reintroduced potentially offering a cure rather than lifelong management.

Lara emphasized that these advances allow children to live longer, healthier, and more fulfilling lives transforming not just patient outcomes but the entire future of pediatric hematology and oncology.

She concluded by expressing her excitement about the collaboration and knowledge exchange made possible by international conferences like this one.

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