Aim: Polymersomes are created to deliver an enzyme-based therapy to the brain in lysosomal storage disease patients. Materials & methods: Polymersomes are formed via the injection method using poly(ethylene glycol)-b-poly(lactic acid) (PEGPLA) and bound to apolipoprotein E, to create a brain-targeted delivery vehicle. Results: Polymersomes have a smallest average diameter of 145 ± 21 nm and encapsulate β-galactosidase at 72.0 ± 12.2% efficiency. PEGPLA polymersomes demonstrate limited release at physiologic pH (7.4), with a burst release at the acidic pH (4.8) of the lysosome. PEGPLA polymersomes facilitate delivery of active β-galactosidase to an in vitro model of GM1 gangliosidosis. Conclusion: The foundation has been laid for testing of PEGPLA polymersomes to deliver enzymatic treatments to the brain in lysosomal storage disorders for the first time.
All Science Journal Classification (ASJC) codes
- Medicine (miscellaneous)
- Biomedical Engineering
- Materials Science(all)