Application of a Quality-By-Design Approach to Optimise Lipid-Polymer Hybrid Nanoparticles Loaded with a Splice-Correction Antisense Oligonucleotide: Maximising Loading and Intracellular Delivery.

Thanki, Kaushik; Pápai, Simon; Lokras, Abhijeet; Rose, Fabrice; Falkenberg, Emily; Franzyk, Henrik; Foged, Camilla

doi:10.1007/s11095-018-2566-3

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Application of a Quality-By-Design Approach to Optimise Lipid-Polymer Hybrid Nanoparticles Loaded with a Splice-Correction Antisense Oligonucleotide: Maximising Loading and Intracellular Delivery.

Author(s): Kaushik Thanki ¹ , Simon Papai ¹ , Abhijeet Lokras ¹ , Fabrice Rose ¹ , Emily Falkenberg ¹ , Henrik Franzyk ² , Camilla Foged ³

Publication date (Electronic): Jan 09 2019

Journal: Pharmaceutical research

Publisher: Springer Science and Business Media LLC

Keywords: HeLa pLuc/705 cells, antisense oligonucleotides, in vitro splice correction, lipid-polymer hybrid nanoparticles (LPNs), lipidoids, quality-by-design, statistical optimisation

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Abstract

Antisense oligonucleotides (ASOs) are promising therapeutics for specific modulation of cellular RNA function. However, ASO efficacy is compromised by inefficient intracellular delivery. Lipid-polymer hybrid nanoparticles (LPNs) are attractive mediators of intracellular ASO delivery due to favorable colloidal stability and sustained release properties.

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Journal ID (iso-abbrev): Pharm Res

Title: Pharmaceutical research

Publisher: Springer Science and Business Media LLC

ISSN (Electronic): 1573-904X

ISSN (Print): 0724-8741

Publication date (Electronic): Jan 09 2019

Volume: 36

Issue: 3

Affiliations

[1 ] Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.

[2 ] Department of Drug Design and Pharmacology, Faculty of Health and Medical Sciences,, University of Copenhagen, Jagtvej 162, DK-2100, Copenhagen Ø, Denmark.

[3 ] Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark. camilla.foged@sund.ku.dk.

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Publisher Item ID: 10.1007/s11095-018-2566-3

DOI: 10.1007/s11095-018-2566-3

PubMed ID: 30623253

SO-VID: 6e831d07-7777-4e66-9927-be2c21c17b9d

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Keywords: HeLa pLuc/705 cells,antisense oligonucleotides,in vitro splice correction,lipid-polymer hybrid nanoparticles (LPNs),lipidoids,quality-by-design,statistical optimisation

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Keywords: HeLa pLuc/705 cells, antisense oligonucleotides, in vitro splice correction, lipid-polymer hybrid nanoparticles (LPNs), lipidoids, quality-by-design, statistical optimisation

Application of a Quality-By-Design Approach to Optimise Lipid-Polymer Hybrid Nanoparticles Loaded with a Splice-Correction Antisense Oligonucleotide: Maximising Loading and Intracellular Delivery.

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