A hybrid siRNA delivery complex for enhanced brain penetration and precise amyloid plaque targeting in Alzheimer’s disease mice


BIBLIOGRAPHIC THERAPEUTIC AGENT ANIMAL MODEL EXPERIMENTAL DESIGN OUTCOMES

Bibliographic

Year of Publication:
2017
Contact PI Name:
Qizhi Zhang
Contact PI Affiliation:
Department of Pharmaceutics, School of Pharmacy, Fudan University, Shanghai, China
Co-Authors:
Xiaoyao Zheng, Xiaoying Pang, Peng Yang, Xu Wan, Yue Wei, Qian Guo, Xinguo Jiang
Primary Reference (PubMED ID):
Funding Source:
National Natural Science Foundation of China
National Basic Research Program of China
Study Goal and Principal Findings:

To realize the therapeutic potential of gene drugs for Alzheimer’s disease (AD), non-invasive, tissue specific and efficient delivery technologies must be developed. Here, a hybrid system for amyloid plaques targeted siRNA delivery was formed by PEGylated Poly(2-(N,N-dimethylamino) ethyl methacrylate) (PEG-PDMAEMA) conjugated with two d-peptides, a CGN for brain penetration and a QSH for β-amyloid binding. The hybrid complex CQ/siRNA, composed of 25% MPEG-PDMAEMA, 50% CGN-PEGPDMAEMA and 25% QSH-PEG-PDMAEMA, showed negligible cytotoxicity and could protect siRNA from enzyme degradation. Being taken up by neuron cells, the complexes could escape from lysosomes, release siRNA in the cytoplasm and thus producing effective gene silence (down-regulated protein level to 18.5%). After intravenous injection, CQ/siRNA penetrated into the brain in an intact form and located around the plaques in transgenic AD mice. The precisely amyloid plaques delivery resulted in increased therapeutic activities, which was demonstrated by the strong mRNA (36.4%) knockdown of BACE1 (a therapeutic target of AD), the less yield of enzyme-digested products sAPPb (-42.6%), as well as the better neurons protection than the single component complexes. In conclusion, the hybrid complex could efficiently and precisely deliver an siRNA to the AD lesion and might be a potential candidate for gene therapy for AD.

Therapeutic Agent

Therapeutic Information:
Therapy Type:
Biologic - RNA
Therapeutic Agent:
Hybrid CQ/siRNA
Therapeutic Target:
BACE1

Animal Model

Model Information:
Species:
Mouse
Model Type:
Non-transgenic
Strain/Genetic Background:
CD-1
Species:
Mouse
Model Type:
Non-transgenic
Strain/Genetic Background:
Ha/ICR
Species:
Mouse
Model Type:
APPxPS1
Strain/Genetic Background:
Not Reported
Species:
Mouse
Model Type:
Non-transgenic
Strain/Genetic Background:
Nude

Experimental Design

Is the following information reported in the study?:
Power/Sample Size Calculation
Randomized into Groups
Blinded for Treatment
Blinded for Outcome Measures
Pharmacokinetic Measures
Pharmacodynamic Measures
Toxicology Measures
ADME Measures
Biomarkers
Dose
Formulation
Route of Delivery
Duration of Treatment
Frequency of Administration
Age of Animal at the Beginning of Treatment
Age of Animal at the End of Treatment
Sex as a Biological Variable
Study Balanced for Sex as a Biological Variable
Number of Premature Deaths
Number of Excluded Animals
Statistical Plan
Genetic Background
Inclusion/Exclusion Criteria Included
Conflict of Interest

Outcomes

Outcome Measured
Outcome Parameters
Histopathology
Neuronal Loss
Biochemical
RNase A Degradation
beta-Site Amyloid Precursor Protein Cleaving Enzyme 1 (BACE1)
Soluble Amyloid Precursor Protein beta (sAPP beta)
beta Amyloid Aggregation
Immunochemistry
Brain-beta Amyloid Deposits
Cell Biology
Cell Viability
Cell Uptake
Cytotoxicity
Pharmacokinetics
Blood Brain Barrier Penetration
Drug Concentration-Brain
Pharmacodynamics
Target Engagement (Reduction beta Amyloid Deposits)
Target Engagement (Knockdown BACE1)
Toxicology
Cell Viability
ADME
Cell Distribution
Cell Release

Source URL: http://alzped.nia.nih.gov/hybrid-sirna-delivery-complex