Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies

被引：40

作者：

Fernandez, Elena Fernandez ^{[1
]}

Santos-Carballal, Beatriz ^{[2
]}

de Santi, Chiara ^{[1
]}

Ramsey, Joanne M. ^{[3
]}

MacLoughlin, Ronan ^{[3
,4
,5
]}

Cryan, Sally-Ann ^{[3
]}

Greene, Catherine M. ^{[1
]}

机构：

[1] Beaumont Hosp, Royal Coll Surg Ireland, Dept Clin Microbiol, Lung Biol Grp, Dublin 9, Ireland

[2] ChiPro GmbH, Anne Conway Str 1, D-28359 Bremen, Germany

[3] Royal Coll Surgeons Ireland, Sch Pharm, Dublin 2, Ireland

[4] Trinity Coll Dublin, Sch Pharm & Pharmaceut Sci, Dublin 2, Ireland

[5] Aerogen Ltd, Galway Business Pk, Galway H91 HE94, Ireland

来源：

MATERIALS | 2018年 / 11卷 / 01期

关键词：

cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); lung gene delivery; nanoparticles; biopolymers; PLGA; chitosan; Locked-Nucleic Acid (LNA); TRANSMEMBRANE CONDUCTANCE REGULATOR; DRY POWDER FORMULATIONS; DRUG-DELIVERY; PARTICLE-SIZE; CELL THERAPY; IN-VITRO; CHITOSAN; SYSTEMS; DISEASE; SIRNA;

D O I：

10.3390/ma11010122

中图分类号：

O64 [物理化学（理论化学）、化学物理学];

学科分类号：

070304 ; 081704 ;

摘要：

Lung gene therapy for cystic fibrosis disease has not been successful due to several challenges such as the absence of an appropriate vector. Therefore, optimal delivery of emerging therapeutics to airway epithelial cells demands suitable non-viral systems. In this work, we describe the formulation and the physicochemical investigation of biocompatible and biodegradable polymeric nanoparticles (NPs), including PLGA and chitosan (animal and non-animal), as novel methods for the safe and efficient delivery of CFTR-specific locked nucleic acids (LNAs).

引用

页数：15

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