Zobrazeno 1 - 10
of 131
pro vyhledávání: '"Ronald J. Mandel"'
Autor:
Fredric P. Manfredsson, Nicole K. Polinski, Thyagarajan Subramanian, Nicholas Boulis, Dustin R. Wakeman, Ronald J. Mandel
Publikováno v:
Frontiers in Aging Neuroscience, Vol 12 (2020)
Externí odkaz:
https://doaj.org/article/e1776ee5c46742d6bccf0fa8834b726c
Autor:
Ronald J. Mandel, David J. Marmion, Deniz Kirik, Yaping Chu, Clifford Heindel, Thomas McCown, Steven J. Gray, Jeffrey H. Kordower
Publikováno v:
Acta Neuropathologica Communications, Vol 5, Iss 1, Pp 1-15 (2017)
Abstract Multiple system atrophy (MSA) is a horrible and unrelenting neurodegenerative disorder with an uncertain etiology and pathophysiology. MSA is a unique proteinopathy in which alpha-synuclein (α-syn) accumulates preferentially in oligodendrog
Externí odkaz:
https://doaj.org/article/7865c54b37d840c5a5892613110d3019
Publikováno v:
Neurobiology of Disease, Vol 48, Iss 2, Pp 212-221 (2012)
The field of in vivo gene therapy has matured to the point where there are numerous clinical trials underway including late-stage clinical trials. Several viral vectors are especially efficient and support lifetime protein expression in the brain and
Externí odkaz:
https://doaj.org/article/72be0b7447cd46b1949fdfa6a91dd412
Publikováno v:
Neurobiology of Disease, Vol 29, Iss 3, Pp 446-455 (2008)
Gene transfer strategies to reduce levels of mutant huntingtin (mHtt) mRNA and protein by targeting human Htt have shown therapeutic promise in vivo. Previously, we have reported that a specific, adeno-associated viral vector (rAAV)-delivered short-h
Externí odkaz:
https://doaj.org/article/b9ff9b59dbae4af3b27be35340e8eb41
Autor:
Gunnar Andsberg, Zaal Kokaia, Ronald L. Klein, Nicholas Muzyczka, Olle Lindvall, Ronald J. Mandel
Publikováno v:
Neurobiology of Disease, Vol 9, Iss 2, Pp 187-204 (2002)
Nerve growth factor (NGF) and brain-derived neurotrophic factor (BDNF) were continuously delivered to the striatum at biologically active levels via recombinant adeno-associated viral (rAAV) gene transfer 4–5 weeks prior to 30 min of middle cerebra
Externí odkaz:
https://doaj.org/article/4ec2ae1e8aae49d5a848503cfcb63b8e
Autor:
Janine A. Gilkes, Arun Srivastava, Coy D. Heldermon, Brontie N. Herrera, Ronald J. Mandel, Sanford L. Boye, Shannon E. Boye, Benjamin L. Judkins
Publikováno v:
Gene Therapy. 28:447-455
Mucopolysaccharidosis type IIIB (MPS IIIB) is an autosomal recessive lysosomal disease caused by defective production of the enzyme α-N-acetylglucosaminidase. It is characterized by severe and complex central nervous system degeneration. Effective t
Autor:
Timothy J. Ness, Heather L. Gray-Edwards, Michael A. Savage, Liudmyla Kondratova, Joseph Rabinowitz, Oleksandr Kondratov, Edgar Rodriguez-Lebron, Ronald J. Mandel, Kirsten E. Coleman, Robert D. Bell, Paul D. Gamlin, Sergei Zolotukhin
Publikováno v:
Mol Ther
Non-human primates (NHPs) are a preferred animal model for optimizing adeno-associated virus (AAV)-mediated CNS gene delivery protocols before clinical trials. In spite of its inherent appeal, it is challenging to compare different serotypes, deliver
Publikováno v:
The FASEB Journal. 33
Autor:
Jose A. Pino, Julia Buddendorff, Gonzalo E. Torres, Macarena Vergara, Ronald J. Mandel, Habibeh Khoshbouei, Dimitri N. Koutzoumis
Publikováno v:
Experimental Neurology. 325:113159
Parkinson's disease (PD) is a debilitating condition resulting in motor and non-motor symptoms affecting approximately 10 million people worldwide. Currently, there are no pharmacological treatments that can cure the condition or effectively halt its
Autor:
Ronald J. Mandel, Jeffrey H. Kordower, James Stansell, Hemraj B. Dodiya, Deniz Kirik, Anders Björklund
Publikováno v:
Cell transplantation. 18(2)