Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy

Jessica M. Kelly; Amanda L. Gross; Douglas R. Martin; Mark E. Byrne

doi:10.2217/nnm-2017-0221

Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy

Jessica M. Kelly, Amanda L. Gross, Douglas R. Martin, Mark E. Byrne

Research output: Contribution to journal › Article › peer-review

38 Scopus citations

Abstract

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.

Original language	English (US)
Pages (from-to)	2591-2606
Number of pages	16
Journal	Nanomedicine
Volume	12
Issue number	23
DOIs	https://doi.org/10.2217/nnm-2017-0221
State	Published - Dec 2017
Externally published	Yes

All Science Journal Classification (ASJC) codes

Bioengineering
Medicine (miscellaneous)
Biomedical Engineering
General Materials Science
Development

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title = "Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy",

abstract = "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.",

author = "Kelly, {Jessica M.} and Gross, {Amanda L.} and Martin, {Douglas R.} and Byrne, {Mark E.}",

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doi = "10.2217/nnm-2017-0221",

language = "English (US)",

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AU - Gross, Amanda L.

AU - Martin, Douglas R.

AU - Byrne, Mark E.

PY - 2017/12

Y1 - 2017/12

N2 - 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.

AB - 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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Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy

Abstract

All Science Journal Classification (ASJC) codes

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