Maria Frank-Kamenetsky

Wave Life Sciences (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

14h-index6.5kcitations28works12.02yr avg
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Research Topics

Advanced biosensing and bioanalysis techniques(6), RNA and protein synthesis mechanisms(5), RNA Research and Splicing(5), RNA Interference and Gene Delivery(5), RNA modifications and cancer(5)

Publications28 total

Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT
Molecular Therapy — Nucleic Acids·2024· 12 citedOpen Access
Stereopure Oligonucleotides for the Selective Silencing of Mutant Huntingtin (4703)
Neurology·2020· 4 cited
Allele Selective Antisense Oligonucleotides for Reducing Mutant Huntingtin (P1.8-051)
Neurology·2019
Sa1465 - The Pharmacological Properties of APOC3 Antisense Oligonucleotides are Enhanced by Control of Stereochemistry
Gastroenterology·2018
Stereochemistry enhances pharmacological properties of APOC3 antisense oligonucleotides
Journal of Hepatology·2018· 1 cited
Preclinical studies of WVE-210201, an investigational stereopure antisense oligonucleotide in development for the treatment of patients with duchenne muscular dystrophy (DMD)
Journal of the Neurological Sciences·2017· 1 cited
WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration
Neuromuscular Disorders·2017· 4 cited
Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
Nature Biotechnology·2017· 358 cited
Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington’s Disease (S56.001)
Neurology·2017
Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
The Lancet·2013· 571 citedOpen Access
Abstract 75: Somatic Overexpression and Knockdown in Mice to Identify Causal Genes Underlying 26 Lipid-associated Loci
Arteriosclerosis Thrombosis and Vascular Biology·2013
Silencing of Lipid Metabolism Genes through IRE1α-Mediated mRNA Decay Lowers Plasma Lipids in Mice
Cell Metabolism·2012· 270 citedOpen Access
Abstract 67: Phase I Safety, Pharmacokinetic, and Pharmacodynamic Results for ALN-PCS, a Novel RNAi Therapeutic for the Treatment of Hypercholesterolemia
Arteriosclerosis Thrombosis and Vascular Biology·2012· 13 cited
Abstract 165: RNAi Therapeutics Targeting PCSK9 and ANGPTL3 for Mixed Hyperlipidemia
Arteriosclerosis Thrombosis and Vascular Biology·2012
Activation of ER stress and mTORC1 suppresses hepatic sortilin-1 levels in obese mice
Journal of Clinical Investigation·2012· 104 citedOpen Access
The Scap/SREBP Pathway Is Essential for Developing Diabetic Fatty Liver and Carbohydrate-Induced Hypertriglyceridemia in Animals
Cell Metabolism·2012· 311 citedOpen Access
IRE1α activation protects mice against acetaminophen-induced hepatotoxicity
The Journal of Experimental Medicine·2012· 148 citedOpen Access
Abstract # 10083: From Genotype to Phenotype at a Novel Cholesterol and Myocardial Infarction Locus
Circulation·2010
From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locus
Nature·2010· 1.2k citedOpen Access
Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
Molecular Therapy·2010· 1.1k citedOpen Access
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Frequent Co-authors

Kevin Fitzgerald(13), Naoki Iwamoto(9), Chandra Vargeese(9), Stephany M. Standley(8), Victor Koteliansky(7), David Butler(6), Keith Bowman(6), Timothy Racie(5), Akin Akinc(5), Muthiah Manoharan(5), Hailin Yang(5), Vera M. Ruda(4), Daniel J. Rader(4), Martin A. Maier(4), Lubomir V. Nechev(4), Jay D. Horton(4), Genliang Lu(4), Alanna Strong(3), William Cantley(3), Kallanthottathil G. Rajeev(3)