Tag: Understanding Leber Congenital Amaurosis (LCA)
Science
Breakthrough Gene Therapy for Childhood Blindness: How AIPL1 Treatment Restored Sight to a Blind Boy
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In a world where darkness was the only reality, six-year-old Jace Broadbin’s life changed forever in 2020. Diagnosed with Leber congenital amaurosis (LCA), a rare genetic disorder caused by mutations in the AIPL1 gene, Jace became the youngest patient to receive a groundbreaking gene therapy at London’s Great Ormond...