CLEMSON UNIVERSITY
 LARSEN LABORATORY DRUG DELIVERY - BIOMATERIALS - NANOTECHNOLOGY COLLEGE OF ENGINEERING, COMPUTING, AND APPLIED SCIENCE
CHEMICAL AND BIOMOLECULAR ENGINEERING
POLYMERSOME DELIVERY OF CRISPR-BASED THERAPIES FOR BRAIN DISEASES funding sources: Clemson University FELLOWS collaborators: Mark Blenner (Clemson Department of Chemical and Biomolecular Engineering) Angela Alexander-Bryant (Clemson Department of Bioengineering) According to the World Health Organization, neurologic diseases remain one of the world’s largest growing global burdens due in part to the difficulty of overcoming transport across the blood-brain barrier (BBB). However, the PI has obtained evidence that polyethylene glycol-b-polylactic acid based polymersomes are capable of delivering an active enzyme, β-galactosidase, across the BBB to the entirety of a feline brain using an apolipoprotein E (apoE) targeted approach.. This discovery now enables the delivery of alternative enzymes across the BBB to treat the central nervous system, for the first time allowing gene regulation and editing techniques to be applied to the brain in vivo. The implications of this discovery are widespread; however, our team is currently exploring two different gene-based therapy approaches (1) gene editing for the treatment of GM1 gangliosidosis, a lysosomal storage disorder and (2) nucleotide targeted gene regulation for the protection of astronauts from long-term radiation exposure effects.
CONTACT email larsenj at clemson . edu phone 864.656.2621 office 130 Earle Hall
Shoaib Iqbal
Sara Edgecomb
related presentations 1. Edgecomb, S.; Rovero, C.; Larsen, J. Nanoparticle-Mediated Delivery of CRISPR Cas9 Protein Directly to the Brain for Regulating Therapy. Focus on Creative Inquiry, Clemson University, April 2, 2019. 2. Iqbal, S.; Larsen, J.; Alexander-Bryant, A.; Blenner, M. Surface functionalized polymersomes for intracellular delivery of Cas9/gRNA ribonucleoprotein complexes. International Nanomedicine and Drug Delivery Symposium. Boston, MA, September 2019.