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Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. Sardh E, et al. Among authors: fitzgerald k. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838. N Engl J Med. 2019. PMID: 30726693 Clinical Trial.
Lipid-like materials for low-dose, in vivo gene silencing.
Love KT, Mahon KP, Levins CG, Whitehead KA, Querbes W, Dorkin JR, Qin J, Cantley W, Qin LL, Racie T, Frank-Kamenetsky M, Yip KN, Alvarez R, Sah DW, de Fougerolles A, Fitzgerald K, Koteliansky V, Akinc A, Langer R, Anderson DG. Love KT, et al. Among authors: fitzgerald k. Proc Natl Acad Sci U S A. 2010 Feb 2;107(5):1864-9. doi: 10.1073/pnas.0910603106. Epub 2010 Jan 11. Proc Natl Acad Sci U S A. 2010. PMID: 20080679 Free PMC article.
Treatment of erythropoietin deficiency in mice with systemically administered siRNA.
Querbes W, Bogorad RL, Moslehi J, Wong J, Chan AY, Bulgakova E, Kuchimanchi S, Akinc A, Fitzgerald K, Koteliansky V, Kaelin WG Jr. Querbes W, et al. Among authors: fitzgerald k. Blood. 2012 Aug 30;120(9):1916-22. doi: 10.1182/blood-2012-04-423715. Epub 2012 May 18. Blood. 2012. PMID: 22611156 Free PMC article.
Liver as a target for oligonucleotide therapeutics.
Sehgal A, Vaishnaw A, Fitzgerald K. Sehgal A, et al. Among authors: fitzgerald k. J Hepatol. 2013 Dec;59(6):1354-9. doi: 10.1016/j.jhep.2013.05.045. Epub 2013 Jun 12. J Hepatol. 2013. PMID: 23770039 Free article.
Safety and efficacy of RNAi therapy for transthyretin amyloidosis.
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Coelho T, et al. Among authors: fitzgerald k. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760. N Engl J Med. 2013. PMID: 23984729 Free article. Clinical Trial.
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. 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.
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: fitzgerald k. 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.
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: fitzgerald k. Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18. Chembiochem. 2015. PMID: 25786782
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: fitzgerald k. 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.
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: fitzgerald k. 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.
1,256 results