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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: chen q. 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.
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. Butler JS, et al. Among authors: chen q. Amyloid. 2016 Jun;23(2):109-18. doi: 10.3109/13506129.2016.1160882. Epub 2016 Mar 31. Amyloid. 2016. PMID: 27033334 Free PMC article.
Tissue-specific gene silencing monitored in circulating RNA.
Sehgal A, Chen Q, Gibbings D, Sah DW, Bumcrot D. Sehgal A, et al. Among authors: chen q. RNA. 2014 Feb;20(2):143-9. doi: 10.1261/rna.042507.113. Epub 2013 Dec 19. RNA. 2014. PMID: 24355758 Free PMC article.
Rational design of cationic lipids for siRNA delivery.
Semple SC, Akinc A, Chen J, Sandhu AP, Mui BL, Cho CK, Sah DW, Stebbing D, Crosley EJ, Yaworski E, Hafez IM, Dorkin JR, Qin J, Lam K, Rajeev KG, Wong KF, Jeffs LB, Nechev L, Eisenhardt ML, Jayaraman M, Kazem M, Maier MA, Srinivasulu M, Weinstein MJ, Chen Q, Alvarez R, Barros SA, De S, Klimuk SK, Borland T, Kosovrasti V, Cantley WL, Tam YK, Manoharan M, Ciufolini MA, Tracy MA, de Fougerolles A, MacLachlan I, Cullis PR, Madden TD, Hope MJ. Semple SC, et al. Among authors: chen j, chen q. Nat Biotechnol. 2010 Feb;28(2):172-6. doi: 10.1038/nbt.1602. Epub 2010 Jan 17. Nat Biotechnol. 2010. PMID: 20081866
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