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The nigrostriatal dopaminergic system as a preferential target of repeated exposures to combined paraquat and maneb: implications for Parkinsons disease

Liandi, Case Stud

The therapy for this ultra-rare disease uses a similar strategy to Intellias liver-targeting treatments for hATTR and HAE: it consists of a Cas12i2 nuclease and guide RNA encapsulated in a lipid nanoparticle (LNP)