Research Articles
Rewriting the Blueprint: A New Gene Therapy Offers Hope for a Rare Genetic Disease
Groundbreaking gene therapy shows promising results in treating Hurler disease through metabolic correction using hematopoietic stem cells.
Unlocking the Secrets of Viral Vectors: The 2001 Young Investigator Award's Leap in Gene Therapy
Explore how the 2001 Young Investigator Award revolutionized gene therapy through groundbreaking research on viral vectors and AAV genome optimization.
From Lab to Life: How Larger Animals Are Revolutionizing Gene Therapy
Exploring how large animal models are transforming gene therapy development for human genetic diseases through physiological similarities and translational research.