Zobrazeno 1 - 10
of 37
pro vyhledávání: '"Barry, Ticho"'
Autor:
Kian Huat Lim, Zhou Han, Hyun Yong Jeon, Jacob Kach, Enxuan Jing, Sebastien Weyn-Vanhentenryck, Mikaela Downs, Anna Corrionero, Raymond Oh, Juergen Scharner, Aditya Venkatesh, Sophina Ji, Gene Liau, Barry Ticho, Huw Nash, Isabel Aznarez
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-13 (2020)
Restoration of normal gene expression is one way to treat monogenic disorders. Here the authors target naturally occurring non-productive alternative splicing using antisense oligonucleotides to promote the production of functional proteins.
Externí odkaz:
https://doaj.org/article/b4b17242c1b24dc9905cc03dd97077f2
Autor:
Leif Carlsson, Jonathan C. Clarke, Christopher Yen, Francine Gregoire, Tamsin Albery, Martin Billger, Ann-Charlotte Egnell, Li-Ming Gan, Karin Jennbacken, Edvin Johansson, Gunilla Linhardt, Sofia Martinsson, Muhammad Waqas Sadiq, Nevin Witman, Qing-Dong Wang, Chien-Hsi Chen, Yu-Ping Wang, Susan Lin, Barry Ticho, Patrick C.H. Hsieh, Kenneth R. Chien, Regina Fritsche-Danielson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 330-346 (2018)
mRNA can direct dose-dependent protein expression in cardiac muscle without genome integration, but to date has not been shown to improve cardiac function in a safe, clinically applicable way. Herein, we report that a purified and optimized mRNA in a
Externí odkaz:
https://doaj.org/article/323a8ccca84c43bd8436f92356b5feeb
Autor:
Joseph Sullivan, Elaine Wirrell, Kelly G. Knupp, Dillon Chen, Muhammad Zafar, Robert Flamini, James Stutely, Charlene Brathwaite, Pam Ventola, Javier Avendaño, Kimberly A. Parkerson, Nancy Wyant, Barry Ticho
Publikováno v:
Epilepsybehavior : EB. 137
The purpose of this study is to evaluate adaptive functioning and neurodevelopment study assessments in a prospective study of patients with Dravet syndrome (DS). We present 3-month interim adaptive functioning and neurodevelopment data from the pros
Autor:
Huw M. Nash, Barry Ticho, Juergen Scharner, Mikaela Downs, Hyun Yong Jeon, Sebastien M. Weyn-Vanhentenryck, Sophina Ji, Anna Corrionero, Isabel Aznarez, Enxuan Jing, Kian-Huat Lim, Jacob Kach, Aditya Venkatesh, Zhou Han, Raymond S Oh, Gene Liau
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-13 (2020)
Nature Communications
Nature Communications
While most monogenic diseases are caused by loss or reduction of protein function, the need for technologies that can selectively increase levels of protein in native tissues remains. Here we demonstrate that antisense-mediated modulation of pre-mRNA
Autor:
Kenneth R. Chien, Susan Lin, Patrick C.H. Hsieh, Karin Jennbacken, Muhammad Waqas Sadiq, Francine Gregoire, Yu-Ping Wang, Christopher Y.T. Yen, Jonathan Clarke, Leif Carlsson, Gunilla Linhardt, Qing-Dong Wang, Regina Fritsche-Danielson, Li-Ming Gan, Edvin Johansson, Sofia Martinsson, Martin Billger, Ann-Charlotte Egnell, Nevin Witman, Barry Ticho, Tamsin Albery, Chien-Hsi Chen
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss, Pp 330-346 (2018)
mRNA can direct dose-dependent protein expression in cardiac muscle without genome integration, but to date has not been shown to improve cardiac function in a safe, clinically applicable way. Herein, we report that a purified and optimized mRNA in a
Autor:
Leif, Carlsson, Jonathan C, Clarke, Christopher, Yen, Francine, Gregoire, Tamsin, Albery, Martin, Billger, Ann-Charlotte, Egnell, Li-Ming, Gan, Karin, Jennbacken, Edvin, Johansson, Gunilla, Linhardt, Sofia, Martinsson, Muhammad Waqas, Sadiq, Nevin, Witman, Qing-Dong, Wang, Chien-Hsi, Chen, Yu-Ping, Wang, Susan, Lin, Barry, Ticho, Patrick C H, Hsieh, Kenneth R, Chien, Regina, Fritsche-Danielson
Publikováno v:
Molecular Therapy. Methods & Clinical Development
mRNA can direct dose-dependent protein expression in cardiac muscle without genome integration, but to date has not been shown to improve cardiac function in a safe, clinically applicable way. Herein, we report that a purified and optimized mRNA in a
Autor:
Barry Ticho, Eman M. Salah, Aaron Deykin, Eric T. Whalley, Glenn J. Smits, Stevan P. Tofovic, Edwin K. Jackson
Publikováno v:
Journal of Pharmacology and Experimental Therapeutics. 356:333-340
Heart failure with preserved ejection fraction (HFpEF) is prevalent and often accompanied by metabolic syndrome. Current treatment options are limited. Here, we test the hypothesis that combined A1/A2B adenosine receptor blockade is beneficial in obe
Autor:
Patricia Kay-Mugford, Judith Hsia, Christopher O'Connor, Martha Mayo, Afshin Salsali, Li-Ming Gan, Udo Michael Gohring, Barry Ticho, Christopher J. Larson, Marco Metra, Regina Frische-Danielson, Michael van der Laan, Pierrre Mugnier, James E. Udelson, Mihai Gheorghiade, Sekayi Mushonga, John R. Teerlink, Robert J.A. Frost, Joerg Koglin, Carol Satler, Sanjiv J. Shah, Gabriel Brooks, Javed Butler, Lothar Roessig, Mary M. DeSouza, Dalane W. Kitzman, Clyde W. Yancy, Carine E. Hamo, Mark Donovan, Shiro Ishihara, Dahlia Garza, Cesare Russo, Victor Shi, Norman Stockbridge, Christophe Depre, Jennifer Hellawell, Harold S. Bernstein, Wilfried Dinh, Hani N. Sabbah, Marc Kozinn
Publikováno v:
Circulation. Heart failure, vol 10, iss 4
The increasing burden and the continued suboptimal outcomes for patients with heart failure underlines the importance of continued research to develop novel therapeutics for this disorder. This can only be accomplished with successful translation of
Publikováno v:
European Journal of Neurology. 21:299-304
Background and purpose Progressive multifocal leukoencephalopathy (PML) is caused by reactivation of JC virus (JCV) infection due to combined host and viral factors. Anti-JCV antibodies provide a means to assess JCV exposure and stratify PML risk. Th
Autor:
William B. Smith, Steven Goldman, Stuart D. Russell, Douglas Chapman, Barry Ticho, Aaron Deykin, Stephen S. Gottlieb, Ignatius Thomas, David DeNofrio, William T. Abraham
Publikováno v:
The Journal of Clinical Pharmacology. 51:899-907
Previous studies suggest that adenosine A 1 receptor antagonists may promote natriuresis without deleterious effects on renal function. This study evaluated renal and hemodynamic effects as well as safety, pharmacokinetics, and tolerability of BG9928