Gene therapy
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University Hospitals Rainbow Babies & Children’s Sickle Cell Disease Patient to Represent the Hospital in Washington, DC
During the 2024 Children’s Hospital Association Family Advocacy Day Canaan Evans and his mom, Arenda will discuss their experience of participating in a clinical trial and undergoing an investigational treatment he says changed his life.
UH Cleveland Medical Center Enrolls First Patient at the Site for an AAV Gene Therapy Trial Treating OTC Deficiency
Groundbreaking treatment could ultimately lead OTC deficient patients to a normal lifestyle after years of medications, dietary protein restriction and recurrent hospitalizations.
Scientists Successfully Test New Way to Deliver Gene Therapy
Researchers use lipids to safely deliver gene therapy to the eye, successfully holding off advance of rare, inherited eye disorder.
Cleveland Researchers Test Novel Gene Therapy for Glioblastoma
Promising results that led to new $2.7 million NCI grant to be presented at ASCO annual meeting on June 2.