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Items: 1 to 20 of 30

1.

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.

Zimmermann TS, Karsten V, Chan A, Chiesa J, Boyce M, Bettencourt BR, Hutabarat R, Nochur S, Vaishnaw A, Gollob J.

Mol Ther. 2017 Jan 4;25(1):71-78. doi: 10.1016/j.ymthe.2016.10.019. Epub 2017 Jan 4.

2.

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.

N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13.

3.

An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria.

Liebow A, Li X, Racie T, Hettinger J, Bettencourt BR, Najafian N, Haslett P, Fitzgerald K, Holmes RP, Erbe D, Querbes W, Knight J.

J Am Soc Nephrol. 2017 Feb;28(2):494-503. doi: 10.1681/ASN.2016030338. Epub 2016 Jul 18.

4.

Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis.

Butler JS, Chan A, Costelha S, Fishman S, Willoughby JL, Borland TD, Milstein S, Foster DJ, Gonçalves P, Chen Q, Qin J, Bettencourt BR, Sah DW, Alvarez R, Rajeev KG, Manoharan M, Fitzgerald K, Meyers RE, Nochur SV, Saraiva MJ, Zimmermann TS.

Amyloid. 2016 Jun;23(2):109-18. doi: 10.3109/13506129.2016.1160882. Epub 2016 Mar 31.

5.

ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients.

Gottlieb J, Zamora MR, Hodges T, Musk AW, Sommerwerk U, Dilling D, Arcasoy S, DeVincenzo J, Karsten V, Shah S, Bettencourt BR, Cehelsky J, Nochur S, Gollob J, Vaishnaw A, Simon AR, Glanville AR.

J Heart Lung Transplant. 2016 Feb;35(2):213-21. doi: 10.1016/j.healun.2015.08.012. Epub 2015 Sep 3.

PMID:
26452996
6.

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study.

Suhr OB, Coelho T, Buades J, Pouget J, Conceicao I, Berk J, Schmidt H, Waddington-Cruz M, Campistol JM, Bettencourt BR, Vaishnaw A, Gollob J, Adams D.

Orphanet J Rare Dis. 2015 Sep 4;10:109. doi: 10.1186/s13023-015-0326-6.

7.

Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study.

Adams D, Coelho T, Obici L, Merlini G, Mincheva Z, Suanprasert N, Bettencourt BR, Gollob JA, Gandhi PJ, Litchy WJ, Dyck PJ.

Neurology. 2015 Aug 25;85(8):675-82. doi: 10.1212/WNL.0000000000001870. Epub 2015 Jul 24.

8.

Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials.

Suanprasert N, Berk JL, Benson MD, Dyck PJ, Klein CJ, Gollob JA, Bettencourt BR, Karsten V, Dyck PJ.

J Neurol Sci. 2014 Sep 15;344(1-2):121-8. doi: 10.1016/j.jns.2014.06.041. Epub 2014 Jun 27.

PMID:
25012480
9.

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.

Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3.

10.

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.

N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.

11.

An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia.

Schmidt PJ, Toudjarska I, Sendamarai AK, Racie T, Milstein S, Bettencourt BR, Hettinger J, Bumcrot D, Fleming MD.

Blood. 2013 Feb 14;121(7):1200-8. doi: 10.1182/blood-2012-09-453977. Epub 2012 Dec 6.

12.

Organization and evolution of hsp70 clusters strikingly differ in two species of Stratiomyidae (Diptera) inhabiting thermally contrasting environments.

Garbuz DG, Yushenova IA, Zatsepina OG, Przhiboro AA, Bettencourt BR, Evgen'ev MB.

BMC Evol Biol. 2011 Mar 22;11:74. doi: 10.1186/1471-2148-11-74.

13.

Natural variation of the amino-terminal glutamine-rich domain in Drosophila argonaute2 is not associated with developmental defects.

Hain D, Bettencourt BR, Okamura K, Csorba T, Meyer W, Jin Z, Biggerstaff J, Siomi H, Hutvagner G, Lai EC, Welte M, Müller HA.

PLoS One. 2010 Dec 17;5(12):e15264. doi: 10.1371/journal.pone.0015264.

14.

In vivo quantification of formulated and chemically modified small interfering RNA by heating-in-Triton quantitative reverse transcription polymerase chain reaction (HIT qRT-PCR).

Landesman Y, Svrzikapa N, Cognetta A 3rd, Zhang X, Bettencourt BR, Kuchimanchi S, Dufault K, Shaikh S, Gioia M, Akinc A, Hutabarat R, Meyers R.

Silence. 2010 Aug 23;1(1):16. doi: 10.1186/1758-907X-1-16.

15.

Duplicate gene evolution toward multiple fates at the Drosophila melanogaster HIP/HIP-Replacement locus.

Hogan CC, Bettencourt BR.

J Mol Evol. 2009 Apr;68(4):337-50. doi: 10.1007/s00239-009-9213-x. Epub 2009 Mar 31.

PMID:
19333534
16.

Natural variation in Drosophila stressed locomotion meets or exceeds variation caused by hsp70 mutation: analysis of behavior and performance.

Bettencourt BR, Drohan BW, Ireland AT, Santhanam M, Smrtic MB, Sullivan EM.

Behav Genet. 2009 May;39(3):306-20. doi: 10.1007/s10519-009-9256-1. Epub 2009 Feb 26.

PMID:
19242787
18.

Polyglutamine expansion in Drosophila: thermal stress and Hsp70 as selective agents.

Bettencourt BR, Hogan CC, Nimali M.

J Biosci. 2007 Apr;32(3):537-47. Review.

19.
20.

Comparative genome sequencing of Drosophila pseudoobscura: chromosomal, gene, and cis-element evolution.

Richards S, Liu Y, Bettencourt BR, Hradecky P, Letovsky S, Nielsen R, Thornton K, Hubisz MJ, Chen R, Meisel RP, Couronne O, Hua S, Smith MA, Zhang P, Liu J, Bussemaker HJ, van Batenburg MF, Howells SL, Scherer SE, Sodergren E, Matthews BB, Crosby MA, Schroeder AJ, Ortiz-Barrientos D, Rives CM, Metzker ML, Muzny DM, Scott G, Steffen D, Wheeler DA, Worley KC, Havlak P, Durbin KJ, Egan A, Gill R, Hume J, Morgan MB, Miner G, Hamilton C, Huang Y, Waldron L, Verduzco D, Clerc-Blankenburg KP, Dubchak I, Noor MA, Anderson W, White KP, Clark AG, Schaeffer SW, Gelbart W, Weinstock GM, Gibbs RA.

Genome Res. 2005 Jan;15(1):1-18.

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