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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)

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This function can be beneficial for athletes during the pre-competition period, as the use of peptide does not result in linear loss of muscle mass but rather only of fat deposits

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

doi: 10.1213/ANE.0000000000002057 10