Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease.

doi:10.1089/nat.2021.0026

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Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease.

Nucleic acid therapeutics - 1 Oct 2021

Murray Justin K, Long Jason, Liu Lei, Singh Shivani, Pruitt Danielle, Ollmann Michael, Swearingen Elissa, Hardy Miki, Homann Oliver, Wu Bin, Holder Jerry Ryan, Sham Kelvin, Herberich Brad, Lo Mei-Chu, Dou Hui, Shkumatov Artem, Florio Monica, Rulifson Ingrid C

Abstract excerpt

Human genome wide association studies confirm the association of the rs738409 single nucleotide polymorphism (SNP) in the gene encoding protein patatin like phospholipase domain containing 3 (PNPLA3) with nonalcoholic fatty liver disease (NAFLD); the presence of the resulting mutant PNPLA3 I148M protein is a driver of nonalcoholic steatohepatitis (NASH). While Pnpla3-deficient mice do not display an adverse...

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PMID: 34297902
DOI: 10.1089/nat.2021.0026

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