IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production
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Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines
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Antisense STAT3 inhibitor decreases viability of myelodysplastic and leukemic stem cells
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Protein production is an early biomarker for RNA-targeted therapies
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An Investigational RNAi Therapeutic Targeting Factor XII (ALN-F12) for the Treatment of Hereditary Angioedema
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Preclinical development of the TLR9 agonist DV281 as an inhaled aerosolized immunotherapeutic for lung cancer: Pharmacological profile in mice, non-human primates, and human primary cells
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A microRNA-29 mimic (remlarsen) represses extracellular matrix expression and fibroplasia in the skin
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Inferring Half-Lives at the Effect Site of Oligonucleotide Drugs
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Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques
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AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model
Evers MM, Miniarikova J, Juhas S, Vallès A, Bohuslavova B, Juhasova J, Skalnikova HK, Vodicka P, Valekova I, Brouwers C, Blits B, Lubelski J, Kovarova H, Ellederova Z, van Deventer SJ, Petry H, Motlik J, Konstantinova P. Mol Ther. 2018 Sep 5;26(9):2163-2177. PubMed
