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Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy
Jessica M Kelly1,2,3, Amanda L Gross2,4, Douglas R Martin2,3,4
1Biomimetic & Biohybrid Materials, Biomedical Devices, & Drug Delivery Laboratories, Department of Chemical Engineering, Samuel Ginn College of Engineering, Auburn University, Auburn, AL 36849, USA.
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.
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01:24Site-Targeted Drug Delivery Systems: Polymeric Carriers
01:31Lysosomes