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.
基金:
Specialized Research Fund for Doctoral ProgramSpecialized Research Fund for the Doctoral Program of Higher Education (SRFDP) [20090071120066]; Fudan University; Shanghai Municipal Education CommissionShanghai Municipal Education Commission (SHMEC); Shanghai Education Development Foundation; National Key Basic Research Program of China (973 Program)National Basic Research Program of China [2013CB932502]
第一作者单位:[1]Fudan Univ, Key Lab Smart Drug Delivery, Minist Educ, Shanghai 201203, Peoples R China[2]Fudan Univ, Sch Pharm, Dept Pharmaceut, PLA, Shanghai 201203, Peoples R China
通讯作者:
通讯机构:[1]Fudan Univ, Key Lab Smart Drug Delivery, Minist Educ, Shanghai 201203, Peoples R China[2]Fudan Univ, Sch Pharm, Dept Pharmaceut, PLA, Shanghai 201203, Peoples R China[*1]Fudan Univ, Key Lab Smart Drug Delivery, Minist Educ, 826 Zhangheng Rd, Shanghai 201203, Peoples R China
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.doi:10.1007/s11095-013-1005-8.
APA:
Huang, Rongqin,Ma, Haojun,Guo, Yubo,Liu, Shuhuan,Kuang, Yuyang...&Jiang, Chen.(2013).Angiopep-Conjugated Nanoparticles for Targeted Long-Term Gene Therapy of Parkinson's Disease.PHARMACEUTICAL RESEARCH,30,(10)
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