Zobrazeno 1 - 10
of 27
pro vyhledávání: '"Scott A. Barros"'
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
Autor:
Sean C. Semple, Xinyao Du, Barbara L. Mui, Akin Akinc, Muthusamy Jayaraman, Roy van der Meel, Jayesh A. Kulkarni, Martin Maier, Thomas D. Madden, Scott A Barros, Kevin Fitzgerald, Pieter R. Cullis, Muthiah Manoharan, Steven M. Ansell, Marco A. Ciufolini, Ying K. Tam, Michael J. Hope, Dominik Witzigmann
Publikováno v:
Nature Nanotechnology, 14(12), 1084-1087. Nature Publishing Group
The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therap
Autor:
Krishna Aluri, Laura Sepp-Lorenzino, Christopher R. Brown, Kevin Fitzgerald, Akin Akinc, Martin Maier, Lauren Blair, Peter F Smith, Ju Liu, Jing Li, Ramesh Indrakanti, Biplab Das, Xiumin Liu, Ivan Zlatev, Scott A Barros, Kallanthottathil G. Rajeev, Yongfeng Jiang, Saket Agarwal, Akshay Vaishnaw, Chris Tran, Klaus Charisse, Jingxuan Liu, Muthiah Manoharan, Shigeo Matsuda, Jayaprakash K. Nair, Jessica E. Sutherland, Tracy Zimmermann, Yuanxin Xu, Jing-Tao Wu, Wendell P Davis, Xuemei Zhang, Maja M. Janas, Vasant Jadhav, Mark K Schlegel
Publikováno v:
Nucleic Acids Research
For oligonucleotide therapeutics, chemical modifications of the sugar-phosphate backbone are frequently used to confer drug-like properties. Because 2′-deoxy-2′-fluoro (2′-F) nucleotides are not known to occur naturally, their safety profile wa
Autor:
Varun Kumar, June Qin, Yongfeng Jiang, Richard G Duncan, Benjamin Brigham, Shannon Fishman, Jayaprakash K Nair, Akin Akinc, Scott A Barros, Pia V Kasperkovitz
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 3, Iss C (2014)
Formulation of short interfering RNA (siRNA) into multicomponent lipid nanoparticles (LNP) is an effective strategy for hepatic delivery and therapeutic gene silencing. This study systematically evaluated the effect of polyethylene glycol (PEG) densi
Externí odkaz:
https://doaj.org/article/91eeff39ed3543dba1ef6fa9a40786ba
Autor:
Scott Waldron, Satya Kuchimanchi, Scott A Barros, Maja M. Janas, Yongfeng Jiang, Mark K Schlegel
Publikováno v:
Nucleic Acid Therapeutics. 27:11-22
Single-stranded (ss) 2'-fluoro (2'-F)-modified oligonucleotides (ONs) with a full phosphorothioate (PS) backbone have been reported to be cytotoxic and cause DNA double-strand breaks (DSBs) when transfected into HeLa cells. However, the molecular det
Autor:
Richard G Duncan, Maja M. Janas, Yongfeng Jiang, Antoinette N Hayes, Pia V Kasperkovitz, Ju Liu, Michael E Placke, Scott A Barros
Publikováno v:
Nucleic Acid Therapeutics. 26:363-371
Registration of pharmaceuticals requires an assessment of their genotoxic potential using in vitro and in vivo tests outlined in the International Conference on Harmonisation (ICH) guidance S2(R1). We have evaluated numerous siRNA-N-acetylgalactosami
Autor:
Zhanna Sobol, Catherine C. Priestley, Michael J. Schlosser, Kevin S. Sweder, Scott A Barros, Frederick B. Oleson, Cindy L Berman, Peter Kasper, Sheila M. Galloway
Publikováno v:
Nucleic Acid Therapeutics. 26:73-85
The Oligonucleotide Safety Working Group subcommittee on genotoxicity testing considers therapeutic oligonucleotides (ONs) unlikely to be genotoxic based on their properties and on the negative results for ONs tested to date. Nonetheless, the subcomm
Autor:
Michael J. Schlosser, Sheila M. Galloway, Kevin S. Sweder, Peter Kasper, Catherine C. Priestley, Zhanna Sobol, Cindy L Berman, Scott A. Barros, Frederick B. Oleson
Publikováno v:
Oligonucleotide-Based Drugs and Therapeutics
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::907bbe5528a0d99d2f4f0a47f209eca4
https://doi.org/10.1002/9781119070153.ch10
https://doi.org/10.1002/9781119070153.ch10
Autor:
Christopher A. Alabi, Tatiana Novobrantseva, Roman L. Bogorad, William Querbes, Weiheng Wang, Abigail K. R. Lytton-Jean, Daniel G. Anderson, Avi Schroeder, Akin Akinc, Arturo J. Vegas, Hao Yin, Carmen Barnes, Yi Chen, Delai Chen, Kevin Fitzgerald, J. Robert Dorkin, June Qin, Scott A Barros, Victor Koteliansky, Yizhou Dong, Robert Langer, Julia Hettinger, Sasilada Sirirungruang, Gaurav Sahay, Kevin T. Love, Daniel J. Siegwart, Mary Carioto, Varun Kumar, Yunlong Zhang, Karsten Olejnik
Publikováno v:
Proceedings of the National Academy of Sciences. 111:3955-3960
siRNA therapeutics have promise for the treatment of a wide range of genetic disorders. Motivated by lipoproteins, we report lipopeptide nanoparticles as potent and selective siRNA carriers with a wide therapeutic index. Lead material cKK-E12 showed
Autor:
Scott A Barros, Jared Gollob
Publikováno v:
Advanced Drug Delivery Reviews. 64:1730-1737
The emerging class of RNA interference (RNAi) therapeutics is a fundamentally novel approach to treating human disease by enabling the pursuit of molecular targets considered “undruggable” by small molecules and traditional protein therapeutics.
Autor:
Scott A Barros, Gregory Hinkle
Publikováno v:
Drug Discovery Toxicology
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::fa9054cc106eb0d7280f51bb22ade7e8
https://doi.org/10.1002/9781119053248.ch4
https://doi.org/10.1002/9781119053248.ch4