The Serum-Resistant Transfection Evaluation and Long-Term Stability of Gene Delivery Dry Powder Based on Mesoporous Silica Nanoparticles and Polyethyleneimine by Freezing-Drying

Mesoporous silica nanoparticles (MSNs) with large surface area, tunable pore size, and low toxicity can act as suitable vehicles for drug and gene delivery. An MSN/DNA/PEI complex delivery system was prepared by using MSNs to hold plasmid DNA coated with polyethyleneimine (PEI), and the dry powder f...

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Bibliographic Details
Published in:AAPS PharmSciTech 2017-07, Vol.18 (5), p.1536-1543
Main Authors: Zhang, Xiaoxu, Zhang, Jianpan, Quan, Guilan, Yang, Pianpian, Pan, Xin, Wu, Chuanbin
Format: Article
Language:English
Subjects:
Animals
Biochemistry
Biomedical and Life Sciences
Biomedicine
Biotechnology
Desiccation - methods
Excipients - chemistry
Excipients - pharmacology
Freeze Drying - methods
Gene Transfer Techniques
Genetic Therapy - instrumentation
Genetic Therapy - methods
Humans
Nanoparticles
Pharmacology/Toxicology
Pharmacy
Plasmids
Polyethyleneimine - chemistry
Polyethyleneimine - pharmacology
Powders
Research Article
Transfection - methods
Trehalose - chemistry
Trehalose - pharmacology
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Summary:Mesoporous silica nanoparticles (MSNs) with large surface area, tunable pore size, and low toxicity can act as suitable vehicles for drug and gene delivery. An MSN/DNA/PEI complex delivery system was prepared by using MSNs to hold plasmid DNA coated with polyethyleneimine (PEI), and the dry powder formulation was produced by freeze-drying with trehalose as lyoprotectant. The MSN/DNA/PEI complexes successfully enhanced the gene expression with about 1.5-fold higher efficiency as compared with the control, and even better effects and lower toxicity were achieved at lower content of PEI. Also, this gene delivery system showed nearly sixfold higher efficiency in the serum-containing condition than the control, so further application of these vehicles in vivo is highly appreciated. Besides, the trehalose containing lyophilized formulation could hold the availability for at least 4 months of storing at room temperature, presenting the potential for industrial production and transportation of gene therapy.
ISSN:1530-9932
1530-9932
DOI:10.1208/s12249-016-0617-9