Zobrazeno 1 - 10
of 29
pro vyhledávání: '"Jay S, Charleston"'
Autor:
Travis K Warren, Chris A Whitehouse, Jay Wells, Lisa Welch, Jay S Charleston, Alison Heald, Donald K Nichols, Marc E Mattix, Gustavo Palacios, Jeffrey R Kugleman, Patrick L Iversen, Sina Bavari
Publikováno v:
PLoS Neglected Tropical Diseases, Vol 10, Iss 2, p e0004456 (2016)
Marburg virus (MARV) is an Ebola-like virus in the family Filovirdae that causes sporadic outbreaks of severe hemorrhagic fever with a case fatality rate as high as 90%. AVI-7288, a positively charged antisense phosphorodiamidate morpholino oligomer
Externí odkaz:
https://doaj.org/article/c8b3af9a51de44cb95f7da37c00b05f2
Autor:
Travis K. Warren, Chris A. Whitehouse, Jay Wells, Lisa Welch, Alison E. Heald, Jay S. Charleston, Pete Sazani, St. Patrick Reid, Patrick L. Iversen, Sina Bavari
Publikováno v:
mBio, Vol 6, Iss 1 (2015)
ABSTRACT Ebola viruses (EBOV) cause severe disease in humans and nonhuman primates with high mortality rates and continue to emerge in new geographic locations, including several countries in West Africa, the site of a large ongoing outbreak. Phospho
Externí odkaz:
https://doaj.org/article/19d856969d434bf7a60886a465bb16a6
Autor:
Diane E, Frank, Frederick J, Schnell, Cody, Akana, Saleh H, El-Husayni, Cody A, Desjardins, Jennifer, Morgan, Jay S, Charleston, Valentina, Sardone, Joana, Domingos, George, Dickson, Volker, Straub, Michela, Guglieri, Eugenio, Mercuri, Laurent, Servais, Francesco, Muntoni, Shande, Tang
Publikováno v:
Neurology
ObjectiveTo report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.MethodsPart 1 was a randomized, double-blind, placebo-controll
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9c84c101e1b3dd57b3ae451d3f1d9037
https://ora.ox.ac.uk/objects/uuid:a45c718a-1957-463b-bd36-efe91be71e85
https://ora.ox.ac.uk/objects/uuid:a45c718a-1957-463b-bd36-efe91be71e85
Autor:
Johannes Dworzak, Daniel G. Rudmann, Famke Aeffner, Kristin Wilson, Crystal Faelan, Suzanne Kanaly, Manish Ranjitkar, Holger Lange, J Kris Piper, Steven A. Moore, Joshua C. Black, Anthony J. Milici, D. Frank, Jay S. Charleston, G. David Young, Alexander Moody
Publikováno v:
Archives of Pathology & Laboratory Medicine. 143:197-205
Context.—Duchenne muscular dystrophy is a rare, progressive, and fatal neuromuscular disease caused by dystrophin protein loss. Common investigational treatment approaches aim at increasing dystrophin expression in diseased muscle. Some clinical tr
Autor:
Frederick J. Schnell, Amy Flynt, Jessie Lynch, Helen Eliopoulos, Cas Donoghue, Linda Lowes, Jerry R. Mendell, Anthony J. Milici, Emily Naughton, D. Frank, Sarah Lewis, Johannes Dworzak, Karin Lucas, Zarife Sahenk, Robert Dracker, Linda H. Cripe, Jay S. Charleston, G. David Young, Anne M. Connolly, Lindsay N. Alfano, Louise R. Rodino-Klapac
Publikováno v:
Medicine
This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with
Autor:
Courtney Shanks, Jianbo Zhang, Mark G. Mense, Alok K. Sharma, Michael P. Carver, Harjeet Kaur, Jay S. Charleston, Peter Sazani
Publikováno v:
Journal of Neuromuscular Diseases. 3:381-393
Background Phosphorodiamidate morpholino oligomers (PMOs) are a class of exon skipping drugs including eteplirsen, which has shown considerable promise for treatment of the degenerative neuromuscular disease, Duchenne musculardystrophy (DMD). Objecti
Autor:
Kelly L. Warfield, Peter Sazani, Jay S. Charleston, Sina Bavari, Edward M. Kaye, Travis K. Warren, Alison E. Heald, Jay B. Saoud, Michael Wong, Lisa S. Welch, Jay Wells, Dana L. Swenson, Mohamed Al-Ibrahim, Patrick L. Iversen, Diane Berry
Publikováno v:
New England Journal of Medicine. 373:339-348
AVI-7288 is a phosphorodiamidate morpholino oligomer with positive charges that targets the viral messenger RNA that encodes Marburg virus (MARV) nucleoprotein. Its safety in humans is undetermined.We assessed the efficacy of AVI-7288 in a series of
Autor:
Alison E. Heald, Jay B. Saoud, Edward M. Kaye, Apinya Vutikullird, William B. Smith, Peter Sazani, Patrick L. Iversen, Jay S. Charleston, Michael Wong, Tim Axtelle
Publikováno v:
Antimicrobial Agents and Chemotherapy. 58:6639-6647
Two identical single-ascending-dose studies evaluated the safety and pharmacokinetics (PK) of AVI-6002 and AVI-6003, two experimental combinations of phosphorodiamidate morpholino oligomers with positive charges (PMO plus ) that target viral mRNA enc
Autor:
Lei Chen, Bruce M. Wentworth, Uditha DeAlwis, G. David Young, Anthony J. Milici, Johannes Dworzak, D. Frank, Sarah Lewis, Frederick J. Schnell, Jerry R. Mendell, Jay S. Charleston, Louise R. Rodino-Klapac, Zarife Sahenk, Jon Voss, Cas Donoghue
Publikováno v:
Neurology. 90(24)
ObjectiveTo describe the quantification of novel dystrophin production in patients with Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen.MethodsClinical study 202 was an observational, open-label extension of the randomized
Autor:
Jay S. Charleston, G. David Young, Kristin Wilson, Daniel G. Rudmann, Jon Tinsley, Crystal Faelan, Steven A. Moore, Anthony J. Milici, D. Frank, Janet C. Patterson-Kane
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are neuromuscular disorders that primarily affect boys due to an X-linked mutation in the DMD gene, resulting in reduced to near absence of dystrophin or expression of truncated fo
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cdd011661252ff27fbe18cfc203f5f1e
https://europepmc.org/articles/PMC5788182/
https://europepmc.org/articles/PMC5788182/