Comparison of the expression and toxicity of AAV2/9 carrying the human A53T α-synuclein gene in presence or absence of WPRE

• DOI: 10.1016/j.heliyon.2021.e06302
• Publication Date: 2021-02-18T07:05:55Z
• Authors: Xiuping Sun; Xuan Yu; Ling Zhang; Wenjie Zhao; Manshi Wang; Yu Zhang; Xianglei Li; Ran Gao; Ludivine S. Breger; Sandra Dovero; Gregory Porras; Pierre-Olivier Fe...; Benjamin Dehay; Erwan Bezard; Chuan Qin
• Keywords: Adeno-associated virus; Woodchuck hepatitis virus

Woodchuck Hepatitis Virus Post-transcriptional Regulatory Element (WPRE) is thought to enhance transgene expression of target genes delivered by adeno-associated viral (AAV) vectors. This study assessed the protein α-synuclein, phosphorylated α-synuclein at Serine 129, extent nigrostriatal degeneration as well subsequent behavioral deficits induced unilateral intranigral stereotactic injection in male adult C57BL/6J mice an AAV2/9 expressing A53T human under control synapsin promoter presence or absence WPRE. The WPRE enabled achieve greater and synucleinopathy which was concomitant with worsened forelimb use asymmetry. work refines a mouse Parkinson's disease model anatomo-pathology related deficits.