Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Deborah Fernau"'
Autor:
Brett D. Story, Matthew E. Miller, Allison M. Bradbury, Emily D. Million, Dongsheng Duan, Toloo Taghian, Dominik Faissler, Deborah Fernau, Sidney J. Beecy, Heather L. Gray-Edwards
Publikováno v:
Frontiers in Veterinary Science, Vol 7 (2020)
Mouse models of human disease remain the bread and butter of modern biology and therapeutic discovery. Nonetheless, more often than not mouse models do not reproduce the pathophysiology of the human conditions they are designed to mimic. Naturally oc
Externí odkaz:
https://doaj.org/article/e95df927e9164cefbe9d29cd9a8a6d7c
Autor:
Stephanie G Bertrand, Mohammed Salman Shazeeb, Erin Horn, Deborah Fernau, Miguel Sena-Esteves, Toloo Taghian, Lindsey J Bierfeldt, Aly Abayazeed, Oguz Cataltepe, Stephen Frey, Susan M Tuominen, Jennifer W. Koehler, Heather L. Gray-Edwards, Terence R. Flotte, Matthew J. Gounis
Publikováno v:
Human Gene Therapy. 31:617-625
Thalamic infusion of adeno-associated viral (AAV) vectors has been shown to have therapeutic effects in neuronopathic lysosomal storage diseases. Preclinical studies in sheep model of Tay-Sachs disease demonstrated that bilateral thalamic injections
Autor:
Deborah Fernau, Heather L. Gray-Edwards, Jill Gallagher, Zdenka Ellederova, Neil Aronin, David Howland, Jodi L. McBride, Amanda Taylor, Alison Weiss, Jon D. Hennebold
Publikováno v:
Journal of Huntington's disease
Genetically modified rodent models of Huntington’s disease (HD) have been especially valuable to our understanding of HD pathology and the mechanisms by which the mutant HTT gene alters physiology. However, due to inherent differences in genetics,
Autor:
Jey W. Koehler, Ashley N. Randle, Jillian Gallagher, Amanda L. Gross, Elise B. Diffie, Brett D. Story, Sundeep Chandra, Sara Carl, Kayly Nielsen, Paul Cuddon, Miguel Sena-Esteves, Edwin H. Kolodny, Deborah Fernau, Amanda R. Taylor, Douglas R. Martin, Carly Corado, Xuntian Jiang, Heather L. Gray-Edwards, Annie S. Maguire, Toloo Taghian, Siauna Johnson
Publikováno v:
Mol Genet Metab
Tay-Sachs disease (TSD) is a fatal neurodegenerative disease caused by a deficiency of the enzyme β-N-acetylhexosaminidase A (HexA). TSD naturally occurs in Jacob sheep is the only experimental model of TSD. TSD in sheep recapitulates neurologic fea
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69a0c7cda10ecdd0ead870203e1fd7ba
https://europepmc.org/articles/PMC8811770/
https://europepmc.org/articles/PMC8811770/
Autor:
Sundeep Chandra, Neil Aronin, Ajit S. Puri, Miguel Sena-Esteves, Matthew J. Gounis, Paul D. Gamlin, Heather L. Gray-Edwards, Chris Christou, Elise B. Diffie, Toloo Taghian, Stephanie G Bertrand, Miklos G. Marosfoi, Diane McKenna-Yasek, Deborah Fernau, Ana Rita Batista, Phillip W. L. Tai, Robert M. King, Douglas R. Martin, Tim Kuchel, Oguz Cataltepe, Terence R. Flotte, Anne S Maguire, Raj Perumal
Publikováno v:
Mol Ther
Global gene delivery to the CNS has therapeutic importance for the treatment of neurological disorders that affect the entire CNS. Due to direct contact with the CNS, cerebrospinal fluid (CSF) is an attractive route for CNS gene delivery. A safe and