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127 results

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Page 1
siRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes.
Matsuda S, Keiser K, Nair JK, Charisse K, Manoharan RM, Kretschmer P, Peng CG, V Kel'in A, Kandasamy P, Willoughby JL, Liebow A, Querbes W, Yucius K, Nguyen T, Milstein S, Maier MA, Rajeev KG, Manoharan M. Matsuda S, et al. Among authors: liebow a. ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2. ACS Chem Biol. 2015. PMID: 25730476
Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo.
Rajeev KG, Nair JK, Jayaraman M, Charisse K, Taneja N, O'Shea J, Willoughby JL, Yucius K, Nguyen T, Shulga-Morskaya S, Milstein S, Liebow A, Querbes W, Borodovsky A, Fitzgerald K, Maier MA, Manoharan M. Rajeev KG, et al. Among authors: liebow a. Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18. Chembiochem. 2015. PMID: 25786782
RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice.
Yasuda M, Gan L, Chen B, Kadirvel S, Yu C, Phillips JD, New MI, Liebow A, Fitzgerald K, Querbes W, Desnick RJ. Yasuda M, et al. Among authors: liebow a. Proc Natl Acad Sci U S A. 2014 May 27;111(21):7777-82. doi: 10.1073/pnas.1406228111. Epub 2014 May 12. Proc Natl Acad Sci U S A. 2014. PMID: 24821812 Free PMC article.
Metabolism of (13)C5-hydroxyproline in mouse models of Primary Hyperoxaluria and its inhibition by RNAi therapeutics targeting liver glycolate oxidase and hydroxyproline dehydrogenase.
Li X, Knight J, Fargue S, Buchalski B, Guan Z, Inscho EW, Liebow A, Fitzgerald K, Querbes W, Todd Lowther W, Holmes RP. Li X, et al. Among authors: liebow a. Biochim Biophys Acta. 2016 Feb;1862(2):233-9. doi: 10.1016/j.bbadis.2015.12.001. Epub 2015 Dec 2. Biochim Biophys Acta. 2016. PMID: 26655602 Free PMC article.
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.
Chan A, Liebow A, Yasuda M, Gan L, Racie T, Maier M, Kuchimanchi S, Foster D, Milstein S, Charisse K, Sehgal A, Manoharan M, Meyers R, Fitzgerald K, Simon A, Desnick RJ, Querbes W. Chan A, et al. Among authors: liebow a. Mol Ther Nucleic Acids. 2015 Nov 3;4(11):e263. doi: 10.1038/mtna.2015.36. Mol Ther Nucleic Acids. 2015. PMID: 26528940 Free PMC article.
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.
Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, Nochur SV, Kotelianski V, Horton J, Mant T, Chiesa J, Ritter J, Munisamy M, Vaishnaw AK, Gollob JA, Simon A. Fitzgerald K, et al. Among authors: liebow a. Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3. Lancet. 2014. PMID: 24094767 Free PMC article. Clinical Trial.
127 results