Zobrazeno 1 - 10
of 37
pro vyhledávání: '"Chris B, Russell"'
Autor:
Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B. Russell, Charles A. O'Neill, Erno Pungor, Stuart Bunting, Sylvia Fong
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 142-153 (2022)
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector containing a B-domain-deleted human coagulation factor VIII (hFVIII) gene controlled by a liver-selective promoter. AAV5-hFVIII-SQ is
Externí odkaz:
https://doaj.org/article/76a1d542e7464f39bab1eb5160a97074
Autor:
Yuanbin Ru, Carley Corado, Russell K. Soon Jr, Andrew C. Melton, Adam Harris, Guoying K. Yu, Nancy Pryer, John R. Sinclair, Martin L. Katz, Temitayo Ajayi, David Jacoby, Chris B. Russell, Sanjay Chandriani
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 6, Iss 12, Pp 2437-2447 (2019)
Abstract Objective Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, progressive, fatal neurodegenerative pediatric disorder resulting from deficiencies of the lysosomal enzyme tripeptidyl peptidase 1 that are caused by mutations in TPP
Externí odkaz:
https://doaj.org/article/f1502f91d5a84d26a84b4e9377e1f3b7
Autor:
Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B. Russell, Charles A. O'Neill, Erno Pungor, Stuart Bunting, Sylvia Fong
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 142-153 (2022)
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 142-153 (2022)
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector containing a B-domain-deleted human coagulation factor VIII (hFVIII) gene controlled by a liver-selective promoter. AAV5-hFVIII-SQ is
Autor:
Russell K. Soon, Temitayo Ajayi, Nancy Pryer, Guoying K. Yu, John Sinclair, Sanjay Chandriani, Andrew Melton, Carley R. Corado, Yuanbin Ru, Adam Harris, Chris B. Russell, David Jacoby, Martin L. Katz
Publikováno v:
Annals of Clinical and Translational Neurology
Annals of Clinical and Translational Neurology, Vol 6, Iss 12, Pp 2437-2447 (2019)
Annals of Clinical and Translational Neurology, Vol 6, Iss 12, Pp 2437-2447 (2019)
Objective Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, progressive, fatal neurodegenerative pediatric disorder resulting from deficiencies of the lysosomal enzyme tripeptidyl peptidase 1 that are caused by mutations in TPP1. Identi
Autor:
Sylvia Fong, Bridget Yates, Choong-Ryoul Sihn, Aras N. Mattis, Nina Mitchell, Su Liu, Chris B. Russell, Benjamin Kim, Adebayo Lawal, Savita Rangarajan, Will Lester, Stuart Bunting, Glenn F. Pierce, K. John Pasi, Wing Yen Wong
Publikováno v:
Nature medicine. 28(4)
Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people with severe hemophilia A. Molecular mechanisms underlying sustained AAV5-
Publikováno v:
Journal of neurology. 267(9)
The present study evaluates serum neurofilament light chain (NfL) as a biomarker of disease features in Friedreich’s ataxia (FRDA). NfL levels from serum of 117 subjects (85 FRDA patients, 13 carriers, and 19 controls) were assayed and correlated w
Autor:
Chris B. Russell, Nina Mitchell, K. John Pasi, Savita Rangarajan, Wing Yen Wong, Michael Laffan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Mingjin Li
BACKGROUND: Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ. METHOD
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2fb2f488cde0f386819c674b197bcaa
http://hdl.handle.net/10044/1/75763
http://hdl.handle.net/10044/1/75763
Autor:
Allison M. Bradbury, Patricia O'Donnell, Samantha J. Fisher, Evelyn M. Galban, Oriane Scholler, Jason Pinkstaff, Charles H. Vite, Jessica H. Bagel, Daniel S. Ory, Xuntian Jiang, Carley R. Corado, Chris B. Russell
Publikováno v:
Mol Cell Neurosci
Globoid cell leukodystrophy (GLD, Krabbe disease, Krabbe's disease) is caused by genetic mutations in the gene encoding, galactosylceramidase (GALC). Deficiency of this enzyme results in central and peripheral nervous system pathology, and is charact
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4d296927a98ff9b1e4d179f0dd64941e
https://europepmc.org/articles/PMC7032565/
https://europepmc.org/articles/PMC7032565/
Autor:
David Martin, Ngozi Erondu, Jennifer E. Towne, Greg Kricorian, Chris B. Russell, Ajay Nirula, Alison L. Budelsky, Jon Nilsen, Paul Klekotka
Publikováno v:
Rheumatology. 55:ii43-ii55
IL-17 cytokines are expressed by a variety of cells and mediate host defence against extracellular pathogens. IL-17 is upregulated at sites of inflammation and can synergize with other cytokines, such as TNF-α, to amplify the inflammatory response.
Autor:
Chris B. Russell, Mayte Suárez-Fariñas, Lewis Tomalin, Sandra Garcet, Hanne Norsgaard, David Adrian Ewald, James G. Krueger, Ajay Nirula, Paul Klekotka
Publikováno v:
The Journal of allergy and clinical immunology. 145(3)
Background IL-17 antagonists induce impressive clinical benefits in psoriasis, but it is unknown to what extent cellular and molecular psoriasis characteristics are suppressed by a clinically relevant dose/schedule of any IL-17-receptor antagonist. O