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Muthiah Manoharan
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Silencing of microRNAs in vivo with ‘antagomirs’
J Krützfeldt, N Rajewsky, R Braich, KG Rajeev, T Tuschl, M Manoharan, ...
nature 438 (7068), 685-689, 2005
51232005
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
J Soutschek, A Akinc, B Bramlage, K Charisse, R Constien, M Donoghue, ...
Nature 432 (7014), 173-178, 2004
30042004
Rational design of cationic lipids for siRNA delivery
SC Semple, A Akinc, J Chen, AP Sandhu, BL Mui, CK Cho, DWY Sah, ...
Nature biotechnology 28 (2), 172-176, 2010
19982010
RNAi-mediated gene silencing in non-human primates
TS Zimmermann, ACH Lee, A Akinc, B Bramlage, D Bumcrot, MN Fedoruk, ...
Nature 441 (7089), 111-114, 2006
18982006
Sequence-specific potent induction of IFN-α by short interfering RNA in plasmacytoid dendritic cells through TLR7
V Hornung, M Guenthner-Biller, C Bourquin, A Ablasser, M Schlee, ...
Nature medicine 11 (3), 263-270, 2005
17562005
miR-181a is an intrinsic modulator of T cell sensitivity and selection
QJ Li, J Chau, PJR Ebert, G Sylvester, H Min, G Liu, R Braich, ...
Cell 129 (1), 147-161, 2007
14732007
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics
A Akinc, A Zumbuehl, M Goldberg, ES Leshchiner, V Busini, N Hossain, ...
Nature biotechnology 26 (5), 561-569, 2008
14712008
RNAi therapeutics: a potential new class of pharmaceutical drugs
D Bumcrot, M Manoharan, V Koteliansky, DWY Sah
Nature chemical biology 2 (12), 711-719, 2006
13562006
Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo
M Jayaraman, SM Ansell, BL Mui, YK Tam, J Chen, X Du, D Butler, ...
Angewandte Chemie 124 (34), 8657-8661, 2012
12662012
Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery
H Lee, AKR Lytton-Jean, Y Chen, KT Love, AI Park, ED Karagiannis, ...
Nature nanotechnology 7 (6), 389-393, 2012
12452012
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
A Akinc, MA Maier, M Manoharan, K Fitzgerald, M Jayaraman, S Barros, ...
Nature nanotechnology 14 (12), 1084-1087, 2019
12142019
Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms
A Akinc, W Querbes, S De, J Qin, M Frank-Kamenetsky, KN Jayaprakash, ...
Molecular Therapy 18 (7), 1357-1364, 2010
11762010
Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
C Wolfrum, S Shi, KN Jayaprakash, M Jayaraman, G Wang, RK Pandey, ...
Nature biotechnology 25 (10), 1149-1157, 2007
11432007
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
JK Nair, JLS Willoughby, A Chan, K Charisse, MR Alam, Q Wang, ...
Journal of the American Chemical Society 136 (49), 16958-16961, 2014
10902014
N-2 substituted purines
PD Cook, KS Ramasamy, M Manoharan
US Patent 5,459,255, 1995
9451995
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates
M Frank-Kamenetsky, A Grefhorst, NN Anderson, TS Racie, B Bramlage, ...
Proceedings of the National Academy of Sciences 105 (33), 11915-11920, 2008
8172008
Oligonucleotides containing N-2 substituted purines
PD Cook, KS Ramasamy, M Manoharan
US Patent 5,587,469, 1996
7561996
Substituted purines and oligonucleotide cross-linking
PD Cook, M Manoharan, KS Ramasamy
US Patent 5,681,941, 1997
7171997
A viral microRNA functions as an orthologue of cellular miR-155
E Gottwein, N Mukherjee, C Sachse, C Frenzel, WH Majoros, JTA Chi, ...
Nature 450 (7172), 1096-1099, 2007
7072007
Specificity, duplex degradation and subcellular localization of antagomirs
J Krützfeldt, S Kuwajima, R Braich, KG Rajeev, J Pena, T Tuschl, ...
Nucleic acids research 35 (9), 2885-2892, 2007
6192007
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