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Angiopep-Conjugated Nanoparticles for Targeted Long-Term Gene Therapy of Parkinson's Disease 期刊论文

编号：

fb207236-d8cb-49d7-b800-d7d50c97939d
作者：

Huang, Rongqin(黄容琴)^[1,2]Ma, Haojun^[1,2];Guo, Yubo^[1,2];Liu, Shuhuan^[1,2];Kuang, Yuyang^[1,2];Shao, Kun(邵堃)^[1,2]Li, Jianfeng(李剑峰)^[1,2]Liu, Yang(刘洋)^[1,2]Han, Liang(韩亮)^[1,2]Huang, Shixian^[1,2];An, Sai^[1,2];Ye, Liya^[3];Lou, Jinning^[3];Jiang, Chen(蒋晨)^[1,2]
语种：

English
期刊：

PHARMACEUTICAL RESEARCH ISSN：0724-8741 2013 年 30 卷 10 期 (2549 - 2559) ; OCT
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关键词：

angiopep-conjugated nanoparticles dendrigraft poly-L-lysine gene therapy multiple dosing administrations Parkinson's disease
摘要：

To prepare an angiopep-conjugated dendrigraft poly-L-lysine (DGL)-based gene delivery system and evaluate the neuroprotective effects in the rotenone-induced chronic model of Parkinson's disease (PD). Angiopep was applied as a ligand specifically binding to low-density lipoprotein receptor-related protein (LRP) which is overexpressed on blood-brain barrier (BBB), and conjugated to biodegradable DGL via hydrophilic polyethyleneglycol (PEG), yielding DGL-PEG-angiopep (DPA). In vitro characterization was carried out. The neuroprotective effects were evaluated in a chronic parkinsonian model induced by rotenone using a regimen of multiple dosing intravenous administrations. The successful synthesis of DPA was demonstrated via H-1-NMR. After encapsulating the therapeutic gene encoding human glial cell line-derived neurotrophic factor (hGDNF), DPA/hGDNF NPs showed a sphere-like shape with the size of 119 +/- 12 nm and zeta potential of 8.2 +/- 0.7 mV. Angiopep-conjugated NPs exhibited higher cellular uptake and gene expression in brain cells compared to unmodified counterpart. The pharmacodynamic results showed that rats in the group with five injections of DPA/hGDNF NPs obtained best improved locomotor activity and apparent recovery of dopaminergic neurons compared to those in other groups. This work provides a practical non-viral gene vector for long-term gene therapy of chronic neurodegenerative disorders.
推荐引用方式
GB/T 7714：

Huang Rongqin,Ma Haojun,Guo Yubo, et al. Angiopep-Conjugated Nanoparticles for Targeted Long-Term Gene Therapy of Parkinson's Disease [J].PHARMACEUTICAL RESEARCH,2013,30(10):2549-2559.
APA：

Huang Rongqin,Ma Haojun,Guo Yubo,Liu Shuhuan,&Jiang Chen.(2013).Angiopep-Conjugated Nanoparticles for Targeted Long-Term Gene Therapy of Parkinson's Disease .PHARMACEUTICAL RESEARCH,30(10):2549-2559.
MLA：

Huang Rongqin, et al. "Angiopep-Conjugated Nanoparticles for Targeted Long-Term Gene Therapy of Parkinson's Disease" .PHARMACEUTICAL RESEARCH 30,10(2013):2549-2559.

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