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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: gamba vitalo c. 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.
First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement.
Tabernero J, Shapiro GI, LoRusso PM, Cervantes A, Schwartz GK, Weiss GJ, Paz-Ares L, Cho DC, Infante JR, Alsina M, Gounder MM, Falzone R, Harrop J, White AC, Toudjarska I, Bumcrot D, Meyers RE, Hinkle G, Svrzikapa N, Hutabarat RM, Clausen VA, Cehelsky J, Nochur SV, Gamba-Vitalo C, Vaishnaw AK, Sah DW, Gollob JA, Burris HA 3rd. Tabernero J, et al. Among authors: gamba vitalo c. Cancer Discov. 2013 Apr;3(4):406-17. doi: 10.1158/2159-8290.CD-12-0429. Epub 2013 Jan 28. Cancer Discov. 2013. PMID: 23358650 Clinical Trial.
A status report on RNAi therapeutics.
Vaishnaw AK, Gollob J, Gamba-Vitalo C, Hutabarat R, Sah D, Meyers R, de Fougerolles T, Maraganore J. Vaishnaw AK, et al. Among authors: gamba vitalo c. Silence. 2010 Jul 8;1(1):14. doi: 10.1186/1758-907X-1-14. Silence. 2010. PMID: 20615220 Free PMC article.
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates.
Frank-Kamenetsky M, Grefhorst A, Anderson NN, Racie TS, Bramlage B, Akinc A, Butler D, Charisse K, Dorkin R, Fan Y, Gamba-Vitalo C, Hadwiger P, Jayaraman M, John M, Jayaprakash KN, Maier M, Nechev L, Rajeev KG, Read T, Röhl I, Soutschek J, Tan P, Wong J, Wang G, Zimmermann T, de Fougerolles A, Vornlocher HP, Langer R, Anderson DG, Manoharan M, Koteliansky V, Horton JD, Fitzgerald K. Frank-Kamenetsky M, et al. Among authors: gamba vitalo c. Proc Natl Acad Sci U S A. 2008 Aug 19;105(33):11915-20. doi: 10.1073/pnas.0805434105. Epub 2008 Aug 11. Proc Natl Acad Sci U S A. 2008. PMID: 18695239 Free PMC article.
Development of lipidoid-siRNA formulations for systemic delivery to the liver.
Akinc A, Goldberg M, Qin J, Dorkin JR, Gamba-Vitalo C, Maier M, Jayaprakash KN, Jayaraman M, Rajeev KG, Manoharan M, Koteliansky V, Röhl I, Leshchiner ES, Langer R, Anderson DG. Akinc A, et al. Among authors: gamba vitalo c. Mol Ther. 2009 May;17(5):872-9. doi: 10.1038/mt.2009.36. Epub 2009 Mar 3. Mol Ther. 2009. PMID: 19259063 Free PMC article.
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