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Rare loss of function variants in the hepatokine gene INHBE protect from abdominal obesity.
Deaton AM, Dubey A, Ward LD, Dornbos P, Flannick J; AMP-T2D-GENES Consortium; Yee E, Ticau S, Noetzli L, Parker MM, Hoffing RA, Willis C, Plekan ME, Holleman AM, Hinkle G, Fitzgerald K, Vaishnaw AK, Nioi P. Deaton AM, et al. Among authors: vaishnaw ak. Nat Commun. 2022 Jul 27;13(1):4319. doi: 10.1038/s41467-022-31757-8. Nat Commun. 2022. PMID: 35896531 Free PMC article.
Association of the transthyretin variant V122I with polyneuropathy among individuals of African ancestry.
Parker MM, Damrauer SM, Tcheandjieu C, Erbe D, Aldinc E, Hawkins PN, Gillmore JD, Hull LE, Lynch JA, Joseph J, Ticau S, Flynn-Carroll AO, Deaton AM, Ward LD, Assimes TL, Tsao PS, Chang KM, Rader DJ, Fitzgerald K, Vaishnaw AK, Hinkle G, Nioi P. Parker MM, et al. Among authors: vaishnaw ak. Sci Rep. 2021 Jun 2;11(1):11645. doi: 10.1038/s41598-021-91113-6. Sci Rep. 2021. PMID: 34079032 Free PMC article.
Liver as a target for oligonucleotide therapeutics.
Sehgal A, Vaishnaw A, Fitzgerald K. Sehgal A, et al. 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.
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: vaishnaw ak. 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.
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald K, Vaishnaw AK, Smith PF, Manoharan M, Jadhav V, Wu JT, Maier MA. Janas MM, et al. Among authors: vaishnaw ak. Nucleic Acids Res. 2019 Apr 23;47(7):3306-3320. doi: 10.1093/nar/gkz140. Nucleic Acids Res. 2019. PMID: 30820542 Free PMC article.
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. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838. N Engl J Med. 2019. PMID: 30726693 Clinical Trial.
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.
Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. Fitzgerald K, et al. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13. N Engl J Med. 2017. PMID: 27959715 Free PMC article. Clinical Trial.
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
Solomon SD, Adams D, Kristen A, Grogan M, González-Duarte A, Maurer MS, Merlini G, Damy T, Slama MS, Brannagan TH 3rd, Dispenzieri A, Berk JL, Shah AM, Garg P, Vaishnaw A, Karsten V, Chen J, Gollob J, Vest J, Suhr O. Solomon SD, et al. Circulation. 2019 Jan 22;139(4):431-443. doi: 10.1161/CIRCULATIONAHA.118.035831. Circulation. 2019. PMID: 30586695 Clinical Trial.
48 results