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Molecular Therapy. Nucleic Acids|July 19, 2024
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTTNaoki Iwamoto, Yuanjing Liu, Maria Frank-Kamenetsky, et al.Nature|August 6, 2010
From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locusKiran Musunuru, Alanna Strong, Maria Frank-Kamenetsky, et al.Proceedings of the National Academy of Sciences of the United States of America|August 13, 2008
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primatesMaria Frank-Kamenetsky, Aldo Grefhorst, Norma N Anderson, et al.Pageof 2