Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Cideciyan AV, Jacobson SG, Drack AV, Ho AC, Charng J, Garafalo AV, Roman AJ, Sumaroka A, Han IC, Hochstedler MD, Pfeifer WL, Sohn EH, Taiel M, Schwartz MR, Biasutto P, Wit W, Cheetham ME, Adamson P, Rodman DM, Platenburg G, Tome MD, Balikova I, Nerinckx ...
Artificial Activation of Escherichia coli mazEF and hipBA Toxin-Antitoxin Systems by Antisense Peptide Nucleic Acids as an Antibacterial Strategy
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Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience
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RNA-Mediated Disease Mechanisms in Neurodegenerative Disorders
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PEPCK1 antisense oligonucleotide prevents adiposity and impairs hepatic glycogen synthesis in high-fat male fed rats
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Hepatocyte Notch activation induces liver fibrosis in nonalcoholic steatohepatitis
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Quantitative fluorescence imaging determines the absolute number of locked nucleic acid oligonucleotides needed for suppression of target gene expression
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Application of ion pair chromatography coupled with mass spectrometry to assess antisense oligonucleotides concentrations in living cells
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Injectability as a function of viscosity and dosing materials for subcutaneous administration
Watt RP, Khatri H, Dibble ARG. Int J Pharm. 2018 Nov 7;554:376-386. PubMed