The research goal of the Pan Laboratory is to combine translational and basic research on virus-mediated, in vivo and ex vivo, gene transfer into stem cells, as well as their potential application for gene therapy of patients with lysosomal storage diseases
(LSDs). Questions such as how to efficiently express the corrective gene, what cells should be appropriate targets for long-term gene transfer, what the best method is for getting a therapeutic gene into the cell, and how to make sure it behaves as expected are the main areas for research. The Pan Lab particularly focuses, among more than 50 LSDs, on the mucopolysaccharidoses (MPS) disorders
, where the manifestations in the central nervous system (CNS) remain untreatable, largely due to the impermeability of the blood-brain barrier (BBB) to large molecules including lysosomal enzymes. Using state-of-art technologies in biochemistry, molecular biology, genetic engineering and vectorology, the Pan Lab has engaged its current projects
on developing novel cell- and gene-based therapeutic approaches for treatment of neuropathic MPS, with the benefits from recent knowledge of stem cell biology. The team attempts to discover noninvasive ways to bypass the BBB for sustained protein delivery across the BBB, which may have broader implications in treating other neurological diseases such as Alzheimer’s disease and stroke. The potential therapeutic benefits of findings originating from in vitro experiments are being further determined by preclinical evaluation in animal disease models.