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Co-editing <i>PINK1</i> and <i>DJ-1</i> genes via AAV-delivered CRISPR/Cas9 system in adult monkey brains elicits classic Parkinsonian phenotypes

2020-09-20

Abstract excerpt

Whether direct manipulation of Parkinson’s disease (PD) risk genes in monkey brain can elicit Parkinsonian phenotypes remains an unsolved issue. Here, we employed an adeno-associated virus (AAV)-delivered CRISPR/Cas9 system to directly co-edit PINK1 and DJ-1 genes in the substantia nigra (SN) region of four adult monkey brains. After the operation, two of the monkeys exhibited all classic PD symptoms, including...

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Identifiers and source

Literature Corpus work
e703233d-81a4-50e3-b442-f0297e49d685
DOI
10.1101/2020.09.19.305003
Open publication

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Co-editing <i>PINK1</i> and <i>DJ-1</i> genes via AAV-delivered CRISPR/Cas9 system in adult monkey brains elicits classic Parkinsonian phenotypesDOI 10.1101/2020.09.19.305003
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