Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Elise B. Diffie"'
Autor:
Heather L. Gray-Edwards, Anne S. Maguire, Nouha Salibi, Lauren E. Ellis, Taylor L. Voss, Elise B. Diffie, Jey Koehler, Ashley N. Randle, Amanda R. Taylor, Brandon L. Brunson, Thomas S. Denney, Ronald J. Beyers, Atoska S. Gentry, Amanda L. Gross, Ana R. Batista, Miguel Sena-Esteves, Douglas R. Martin
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 258-270 (2020)
GM1 gangliosidosis (GM1) is a fatal neurodegenerative lysosomal storage disease that occurs most commonly in young children, with no effective treatment available. Long-term follow-up of GM1 cats treated by bilateral thalamic and deep cerebellar nucl
Externí odkaz:
https://doaj.org/article/e5a002b5c4934f989fe4e2733f002031
Autor:
Ronald J. Beyers, Ana Rita Batista, Heather L. Gray-Edwards, Miguel Sena-Esteves, Thomas S. Denney, Ashley N. Randle, Lauren E. Ellis, Jey W. Koehler, Nouha Salibi, Anne S Maguire, Taylor L. Voss, Amanda L. Gross, Elise B. Diffie, Amanda R. Taylor, Atoska S. Gentry, Douglas R. Martin, Brandon L. Brunson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 258-270 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
GM1 gangliosidosis (GM1) is a fatal neurodegenerative lysosomal storage disease that occurs most commonly in young children, with no effective treatment available. Long-term follow-up of GM1 cats treated by bilateral thalamic and deep cerebellar nucl
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