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of 6
pro vyhledávání: '"Francis Fonyuy Tukov"'
Autor:
Eric W. Johnson, Jeffrey J. Sutherland, Emily Meseck, Cameron McElroy, Deepa H. Chand, Francis Fonyuy Tukov, Eloise Hudry, Kelley Penraat
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 208-219 (2023)
In nonhuman primates (NHPs), adeno-associated virus serotype 9 (AAV9) vectorized gene therapy can cause asymptomatic microscopic injury to dorsal root ganglia (DRG) and trigeminal ganglia (TG) somatosensory neurons, causing neurofilament light chain
Externí odkaz:
https://doaj.org/article/ff357e0c44fd444d815f1c28922732c0
Publikováno v:
Gene Therapy.
Human gene replacement therapies such as onasemnogene abeparvovec (OA) use recombinant adeno-associated virus (rAAV) vectors to treat monogenic disorders. The heart and liver are known target organs of toxicity in animals; with cardiac and hepatic mo
Autor:
Francis Fonyuy Tukov, John W. Day, Richard S. Finkel, Kevin A. Strauss, Deepa H. Chand, Jerry R. Mendell, Aaron Kleyn, Sitra Tauscher-Wisniewski, Sandra P. Reyna, Eugenio Mercuri
Publikováno v:
Drug Safety
Introduction This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy. Objective We comprehensively assessed the safety of
Autor:
Francis Fonyuy Tukov, Deepa H. Chand, Rui Sun, Petra Kaufmann, Aaron Kleyn, Franziska Mohr, Kyle Montgomery, Gerd A. Kullak-Ublick, Sitra Tauscher-Wisniewski, Hugh J. McMillan
Publikováno v:
Journal of Hepatology. 74:560-566
Background & Aims Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed to address SMA's root cause. In pivotal mouse toxicology studies, the liver was
Autor:
Francis Fonyuy Tukov, Keith Mansfield, Mark Milton, Emily Meseck, Kelley Penraat, Deepa Chand, Andreas Hartmann
Publikováno v:
Human gene therapy. 33(13-14)
Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children 2 years. For later-onset patients, intrathecal onasemnogene abeparvovec may be advantageous over intravenous administration. Recently, microscop
Autor:
John W. Day, Jerry R. Mendell, Eugenio Mercuri, Richard S. Finkel, Kevin A. Strauss, Aaron Kleyn, Sitra Tauscher-Wisniewski, Francis Fonyuy Tukov, Sandra P. Reyna, Deepa H. Chand
Publikováno v:
Drug Safety. 45:191-192