Next-generation gene therapy for Stargardt Disease: Enhanced AAV-mediated ABCA4 delivery to restore vision
Dr. Jiang-Hui (Sloan) Wang is an early-career scientist working towards developing next-generation retinal gene therapies for conditions such as Leber congenital amaurosis and Stargardt disease. He trained under a gene therapy pioneer in the University of Massachusetts Chan Medical School and has published his work in several scientific journals since.
Dr. Wang strives to accelerate vision-restoring therapies for inherited retinal diseases. He actively engages with patient communities to bridge the gap between research and patient needs and is committed to ensuring that pre-clinical innovations are translated into real-world solutions, to ensure scientific progress directly uplifts communities affected by retinal blindness worldwide
Dr. Wang’s research focuses on overcoming a major challenge in gene therapy for Stargardt disease: safely and efficiently delivering the large ABCA4 gene to the retina to repair the faulty gene involved in causing Stargardt disease.
Dr. Wang plans to deliver the ABCA4 gene into retinal cells (photoreceptors) using a specially modified virus. Once inside the cell, the new gene will restore the faulty gene into its original, functional form.
Dr. Wang will then test the effectiveness of the ABCA4 gene in treating mouse models with Stargardt disease. Dr Wang’s work has the potential to advance gene therapy technologies and revolutionise treatment options for people affected by Stargardt disease and other retinal conditions.
Published: 11 August, 2025
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