Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Abstract excerpt

Rare monogenic disorders such as lysosomal diseases have been at the forefront in the development of novel treatments where therapeutic options are either limited or unavailable. The increasing number of successful pre-clinical and clinical studies in the last decade demonstrates that gene therapy represents a feasible option to address the unmet medical need of these patients. This article provides a...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cellular transport and secretion
• Share to Lysosomal Storage Disorders Research
• Share to Retinal Development and Disorders