Zobrazeno 1 - 10
of 34
pro vyhledávání: '"Frederick J Schnell"'
Autor:
Shayla K Shorter, Frederick J Schnell, Sean R McMaster, David F Pinelli, Rakieb Andargachew, Brian D Evavold
Publikováno v:
PLoS ONE, Vol 11, Iss 2, p e0149582 (2016)
T cells have the remarkable ability to recognize antigen with great specificity and in turn mount an appropriate and robust immune response. Critical to this process is the initial T cell antigen recognition and subsequent signal transduction events.
Externí odkaz:
https://doaj.org/article/04971c8881384a6b9cd6d6583f852d17
Publikováno v:
PLoS Pathogens, Vol 8, Iss 12, p e1003073 (2012)
The viral family Arenaviridae includes a number of viruses that can cause hemorrhagic fever in humans. Arenavirus infection often involves multiple organs and can lead to capillary instability, impaired hemostasis, and death. Preclinical testing for
Externí odkaz:
https://doaj.org/article/a0fbd6ecf3484caa8908e1d864f4d86b
Autor:
Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 479-492 (2022)
Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression, leading to progressive loss of motor and cardiorespiratory function. Four exon-skipping approaches using antisense phospho
Externí odkaz:
https://doaj.org/article/c58f1dc6613c474f8ea734b55a626d47
Autor:
Steve D. Wilton, Michel Tchan, Sue Fletcher, May T. Aung-Htut, Kristin A. Ham, Frederick J. Schnell, Russell D. Johnsen
Publikováno v:
Scientific Reports, Vol 10, Iss 1, Pp 1-12 (2020)
Scientific Reports
Scientific Reports
Pompe disease is caused by mutations in the GAA gene, resulting in deficient lysosomal acid-α-glucosidase activity in patients, and a progressive decline in mobility and respiratory function. Enzyme replacement therapy is one therapeutic option, but
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::baa4a767db12372d72f63a7d949ec7d6
http://link.springer.com/article/10.1038/s41598-020-63461-2
http://link.springer.com/article/10.1038/s41598-020-63461-2
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:
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a723d5cf41ea1e9e860c4bea7d51813a
https://pubmed.ncbi.nlm.nih.gov/31261494
https://pubmed.ncbi.nlm.nih.gov/31261494
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1ff8a7c05ed4d9b8607d4fa16b0ae489
https://pubmed.ncbi.nlm.nih.gov/30249679
https://pubmed.ncbi.nlm.nih.gov/30249679
Publikováno v:
US Neurology. 15:40
The Duchenne muscular dystrophy community has recently seen the first approved therapy for the restoration of dystrophin, based on its ability to increase levels of dystrophin protein, as determined by western blot. The approval, along with the initi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::bb50ff73d64b5a2576521cd2bd3ebc6b
https://doi.org/10.17925/usn.2019.15.1.40
https://doi.org/10.17925/usn.2019.15.1.40
Autor:
Frederick J. Schnell, Kimberly R. Marshall-Batty, Patrick L. Iversen, David E. Greenberg, Mattie M. McKnight, Bruce L. Geller
Publikováno v:
Journal of Infectious Diseases. 208:1553-1560
Background. Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) are synthetic DNA/RNA analogues that silence expression of specific genes. We studied whether PPMOs targeted to essential genes in Acinetobacter lwoffii and Acinetobacter
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::50606235a4a61be986293d4f81f71532
https://doi.org/10.1093/infdis/jit460
https://doi.org/10.1093/infdis/jit460
Publikováno v:
BioResearch Open Access, Vol 2, Iss 1, Pp 61-66 (2013)
BioResearch Open Access
BioResearch Open Access
Phosphorodiamidate morpholino oligomers (PMO) are neutrally charged, sequence-specific antisense agents that interfere with targeted gene expression. PMO have been shown to be highly specific and potent therapies after cellular uptake, yet methods to
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c315c5a41ab86f4335bb1eeac87c3392
https://www.liebertpub.com/doi/full/10.1089/BIORES.2012.0276
https://www.liebertpub.com/doi/full/10.1089/BIORES.2012.0276