Zobrazeno 1 - 10
of 20
pro vyhledávání: '"Tim Racie"'
Autor:
Amy Chan, Abigail Liebow, Makiko Yasuda, Lin Gan, Tim Racie, Martin Maier, Satya Kuchimanchi, Don Foster, Stuart Milstein, Klaus Charisse, Alfica Sehgal, Muthiah Manoharan, Rachel Meyers, Kevin Fitzgerald, Amy Simon, Robert J Desnick, William Querbes
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 4, Iss C (2015)
The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting or drug exposure can lead to accumulation
Externí odkaz:
https://doaj.org/article/39568a2964f14544a20cb66549c751d3
Autor:
Pachamuthu Kandasamy, Shohei Mori, Shigeo Matsuda, Namrata Erande, Dhrubajyoti Datta, Jennifer L. S. Willoughby, Nate Taneja, Jonathan O’Shea, Anna Bisbe, Rajar M. Manoharan, Kristina Yucius, Tuyen Nguyen, Ramesh Indrakanti, Swati Gupta, Jason A. Gilbert, Tim Racie, Amy Chan, Ju Liu, Renta Hutabarat, Jayaprakash K. Nair, Klaus Charisse, Martin A. Maier, Kallanthottathil G. Rajeev, Martin Egli, Muthiah Manoharan
Publikováno v:
Journal of Medicinal Chemistry. 66:2506-2523
Autor:
Dhrubajyoti Datta, Christopher S. Theile, Kelly Wassarman, June Qin, Tim Racie, Karyn Schmidt, Yongfeng Jiang, Rachel Sigel, Maja M. Janas, Martin Egli, Muthiah Manoharan
Publikováno v:
Chemical Communications. 59:6347-6350
A new class of 5′-morpholino modified nucleoside building blocks was synthesized. Among them, Mo2 exhibited better off-target mitigation for RNAi compared to other analogues Pip and Mo3 and the previously reported morpholino derivative Mo1.
Autor:
Krishna Aluri, Swati Gupta, Sarah Leblanc, Martin Egli, Tim Racie, Muthiah Manoharan, Anna Bisbe, Peter Podbevšek, Vasant Jadhav, John Szeto, Christopher R. Brown, Hartmut Jahns, Ivan Zlatev, Dale C. Guenther, Martin Maier, Pawan Kumar, Adam Castoreno, Janez Plavec, Rohan Degaonkar
Publikováno v:
Nucleic Acids Research
In order to achieve efficient therapeutic post-transcriptional gene-silencing mediated by the RNA interference (RNAi) pathway, small interfering RNAs (siRNAs) must be chemically modified. Several supra-RNA structures, with the potential to stabilize
Autor:
Maja M. Janas, Saeho Chong, Mark K Schlegel, Jason Gilbert, Saket Agarwal, Vasant Jadhav, Dale Gunther, Michelle Jung, Mark Keating, Kelly Wassarman, Shigeo Matsuda, Arlin Rogers, Yongfeng Jiang, Lei Johnson, Elena Castellanos-Rizaldos, Jessica E. Sutherland, Scott Lentini, Urjana Poreci, Klaus Charisse, Jing Li, Anne Kasper, Yesseinia Anglero-Rodriguez, Jeff Rollins, Jing-Tao Wu, Alex Kelin, Sarah Le Blanc Sarah Le Blanc, Diana Cha, Martin E. Maier, Adam Castoreno, Samantha Chigas, Tuyen Nguyen, Muthiah Manoharan, Wendell P Davis, Haiyan Peng, Jayaprakash K. Nair, John Petrulis, Christopher S. Theile, Tyler Chickering, Ivan Zlatev, Tim Racie, Kirk Brown, Christopher R. Brown, Stuart Milstein, Jessica Bombardier, Melissa Mobley, Jeffrey Allen, Raj Maganti, Donald Foster, Kevin Fitzgerald, Jeffrey Kurz, Lauren Blair Woods, Robin McDougall
RNA interference (RNAi) therapeutics are a new class of medicines that can address unmet medical needs by silencing disease-causing gene transcripts. While delivery of short interfering RNAs (siRNAs) to hepatocytes has yielded multiple drug approvals
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::1b912cc876215cb4611322852009dc8f
https://doi.org/10.21203/rs.3.rs-946835/v1
https://doi.org/10.21203/rs.3.rs-946835/v1
Autor:
James Butler, Mark D. Fleming, Shannon Fishman, Paul J. Schmidt, Tim Racie, Julia Hettinger, Kaifeng Liu, Kevin Fitzgerald, Gary A. Visner
Publikováno v:
American Journal of Hematology. 93:745-750
Diminished β-globin synthesis in β-thalassemia is associated with ineffective erythropoiesis, leading to secondary iron overload caused by inappropriately low levels of hepcidin and to splenomegaly in the symptomatic thalassemias. Splenectomy is of
Autor:
Theo J.C. Van Berkel, Kun Qian, Muthiah Manoharan, Kallanthottathil G. Rajeev, Vasant Jadhav, Klaus Charisse, Jayaprakash K. Nair, James Butler, Tracy Zimmermann, Amy Chan, Jennifer L. S. Willoughby, Alfica Sehgal, Kristina Yucius, Tim Racie, Tuyen Nguyen, Svetlana Shulga-Morskaya, Martin Maier
Publikováno v:
Molecular Therapy
The hepatocyte-specific asialoglycoprotein receptor (ASGPR) is an ideal candidate for targeted drug delivery to the liver due to its high capacity for substrate clearance from circulation together with its well-conserved expression and function acros
Publikováno v:
American Journal of Hematology. 90:310-313
β-thalassemias result from diminished β-globin synthesis and are associated with ineffective erythropoiesis and secondary iron overload caused by inappropriately low levels of the iron regulatory hormone hepcidin. The serine protease TMPRSS6 attenu
Autor:
Dganit Danino, Omar F. Khan, Klaus Charisse, Matthew J. Webber, Akin Akinc, Yizhou Dong, Tatiana Novobrantseva, Shan Jiang, Roman L. Bogorad, Vera M. Ruda, Matthias Nahrendorf, Taylor E. Shaw, Dayna K. Mudge, Rubin M. Tuder, Aude Thiriot, Andrew Bader, Daniel G. Anderson, Mark W. Kieran, Hendrik B. Sager, Tim Racie, Christopher G. Levins, Lauren Speciner, Partha Dutta, Ulrich H. von Andrian, Robert Langer, Mario F. Perez, Siddharth Jhunjunwala, Kevin Fitzgerald, Avi Schroeder, Kamaljeet Singh Sandhu, Dipak Panigrahy, James E. Dahlman, Ludmila Abezgauz, Lynelle P. Smith, Hao Yin, Danielle Seedorf, Abigail K. R. Lytton-Jean, Victor Koteliansky, Gaurav Sahay, Carmen M. Barnés, Brian T. Kalish, Yiping Xing, Apeksha Dave
Publikováno v:
PMC
Dysfunctional endothelium contributes to more diseases than any other tissue in the body. Small interfering RNAs (siRNAs) can help in the study and treatment of endothelial cells in vivo by durably silencing multiple genes simultaneously, but efficie
Autor:
Christian Wolfrum, Nikolay V. Dokholyan, Gang Wang, Valerie A. Clausen, Julia Hettinger, Svetlana Shulga Morskaya, Qianfan Wang, Joshua Brodsky, Verbena Kosovrasti, Xuemei Zhang, Dinah W.Y. Sah, Mike Meys, Kallanthottathil G. Rajeev, Yupang Fang, Muthiah Manoharan, Jennifer Sharman, Martin Maier, Feng Ding, Mariano Severgnini, Victor Kotelianski, Lubomir Tchangov, Renta Hutabarat, Chang Geng Peng, Robert Li, Tim Racie, Lubomir Nechev, James Butler, Tomoko Nakayama, Scott A Barros, Alfica Sehgal, Markus Stoffel
Publikováno v:
Molecular Therapy, 20 (8)
Molecular Therapy
MOLECULAR THERAPY
Molecular Therapy
MOLECULAR THERAPY
Therapeutics based on RNA interference (RNAi) have emerged as a potential new class of drugs for treating human disease by silencing the target messenger RNA (mRNA), thereby reducing levels of the corresponding pathogenic protein. The major challenge