Article

Robust nuclease-dead S. aureus dCas9-mediated alpha-synuclein knockdown in substantia nigra in a humanized mouse model of Parkinson’s disease

2023-09-05

Abstract excerpt

Parkinson’s disease (PD) is becoming increasingly prevalent due to an aging society, which places a substantial disease burden on patients and their families and an annual cost estimated at 52 billion dollars. However, no approved disease modulatory therapies that halt disease progression are available. Alpha-synuclein is a critical therapeutic target found in aggregated form in Lewy bodies which is the diagnostic...

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Literature Corpus work: 0e827890-0064-5f51-8bee-ec6bbbc0f2b6
DOI: 10.1101/2023.09.05.556425

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