| Yongwei Shu,Jie Yao,Yang Qu,Jing Zheng,Jing Ding,Lina Zhang,Yefan Wang,Jingyu Zhang,Siqi Tang. Intravenous injection of AAV-PHP.eB across the blood-brain barrier in the adult mouse for central nervous system gene therapy. Oncol Transl Med, 2019, 5: 1-5. |
| Intravenous injection of AAV-PHP.eB across the blood-brain barrier in the adult mouse for central nervous system gene therapy |
| Received:October 24, 2018 Revised:March 14, 2019 |
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| KeyWord:gene therapy; AAV-PHP.eB; blood-brain barrier; regulatory element; noninvasive viral injection |
| Author Name | Affiliation | E-mail | | Yongwei Shu | The Fourth Hospital of Harbin Medical University | Shuyongwei163@163.com | | Jie Yao | East China University of Science and Technology | | | Yang Qu | The Fourth Hospital of Harbin Medical University | | | Jing Zheng | East China University of Science and Technology | | | Jing Ding | The Fourth Hospital of Harbin Medical University | | | Lina Zhang | The Fourth Hospital of Harbin Medical University | | | Yefan Wang | East China University of Science and Technology | | | Jingyu Zhang | The Fourth Hospital of Harbin Medical University | | | Siqi Tang | East China University of Science and Technology | tangsiqi1@hotmail.com |
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| Abstract: |
| Objective To verify the neurotypicality of AAV-PHP.eB after tail vein injection in adult mice and its
efficiency in crossing the blood-brain barrier (BBB).
Methods The rAAV-SYN-GFP plasmid was constructed, and adult C57BL mice were injected with AAVPHP.eB: SYN-GFP in the tail vein (300 nL, virus titer 3 × 109 vg) and in the prefrontal lobe (50 L, virus titer
5 × 1011 vg). The green fluorescent protein (GFP) signal in the brain was observed at two weeks, while the
GFP signal in the peripheral organs was observed at four weeks.
Results Two weeks after tail vein injection, GFP expression was observed throughout the brain,
especially in the cortex, hippocampus, and geniculate nucleus. No GFP signal was observed or detected
by western blotting in the peripheral organs after four weeks. GFP signal was observed mainly at the local
site after prefrontal lobe injection.
Conclusion AAV-PHP.eB: SYN-GFP can effectively cross the BBB in adult mice. Using a neuron-specific
promoter allows exogenous gene expression in neurons; therefore, AAV-PHP.eB can be used as an
effective carrier for studying diseases in the central nervous system (CNS). |
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