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Article

CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease

2023-03-16

Abstract excerpt

<title>Abstract</title><p>Parkinson`s disease (PD) is the second most prevalent neurodegenerative disease, and different gene therapy strategies have been used as experimental treatments. As a proof of concept for the treatment of PD, we used SAM, a CRISPR gene activation system, to activate the endogenous tyrosine hydroxylase gene of astrocytes to produce dopamine (DA) in the striatum of 6-OHDA-lesioned rats. Pot...

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Literature Corpus work
12aa6835-00ce-5b47-bd96-4d2edd979f1e
DOI
10.21203/rs.3.rs-2625741/v1
Open publication

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CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s diseaseDOI 10.21203/rs.3.rs-2625741/v1
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