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; Papai, Simon; Lokras, Abhijeet; Rose, Fabrice; Falkenberg, Emily; Franzyk, Henrik; Foged, Camilla

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

Afiliação

Thanki K; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Papai S; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Lokras A; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Rose F; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Falkenberg E; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Franzyk H; Department of Drug Design and Pharmacology, Faculty of Health and Medical Sciences,, University of Copenhagen, Jagtvej 162, DK-2100, Copenhagen Ø, Denmark.
Foged C; Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark. camilla.foged@sund.ku.dk.

Pharm Res ; 36(3): 37, 2019 Jan 09.

Article em En | MEDLINE | ID: mdl-30623253

Assuntos

Sistemas de Liberação de Medicamentos; Nanopartículas/química; Oligonucleotídeos Antissenso/administração & dosagem; Splicing de RNA/genética; Tecnologia Farmacêutica/métodos; Preparações de Ação Retardada/administração & dosagem; Terapia Genética/métodos; Células HeLa; Humanos; Lipídeos/química; Luciferases/genética; Copolímero de Ácido Poliláctico e Ácido Poliglicólico/química

Palavras-chave

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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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Splicing de RNA / Oligonucleotídeos Antissenso / Tecnologia Farmacêutica / Sistemas de Liberação de Medicamentos / Nanopartículas Idioma: En Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Dinamarca

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