Zobrazeno 1 - 10
of 50
pro vyhledávání: '"F. Kaemmerer"'
Autor:
Cliff Heindel, Karlaina J.L. Osmon, Evan Woodley, John G. Keimel, Patrick Thompson, Subha Karumuthil-Melethil, William F. Kaemmerer, Jagdeep S. Walia, Steven J. Gray
Publikováno v:
Current Gene Therapy. 22:262-276
Background: GM2 gangliosidosis is a neurodegenerative, lysosomal storage disease caused by the deficiency of β-hexosaminidase A enzyme (Hex A), an α/β-subunit heterodimer. A novel variant of the human hexosaminidase α-subunit, coded by HEX M, has
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d9733bb70f07b3fa57ffcce8720e2527
https://doi.org/10.2174/1566523221666210916153051
https://doi.org/10.2174/1566523221666210916153051
Autor:
Richard Grondin, Pei Ge, Qingmin Chen, Jessica E Sutherland, Zhiming Zhang, Don M Gash, David K Stiles, Gregory R Stewart, Dinah W Y Sah, William F Kaemmerer
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 4, Iss C (2015)
One possible treatment for Huntington's disease involves direct infusion of a small, interfering RNA (siRNA) designed to reduce huntingtin expression into brain tissue from a chronically implanted programmable pump. Here, we studied the suppression o
Externí odkaz:
https://doaj.org/article/2560f36a757e4533913335385c14852e
Autor:
Shalini Kot, Violeta Zaric, Patrick Thompson, John G. Keimel, Evan Woodley, Jagdeep S. Walia, Erik Lykken, William F. Kaemmerer, Subha Karumuthil-Melethil, Steven J. Gray, Zhilin Chen
Publikováno v:
International Journal of Molecular Sciences
Volume 22
Issue 13
International Journal of Molecular Sciences, Vol 22, Iss 6751, p 6751 (2021)
Volume 22
Issue 13
International Journal of Molecular Sciences, Vol 22, Iss 6751, p 6751 (2021)
GM2 gangliosidosis disorders are a group of neurodegenerative diseases that result from a functional deficiency of the enzyme β-hexosaminidase A (HexA). HexA consists of an α- and β-subunit
a deficiency in either subunit results in Tay–Sach
a deficiency in either subunit results in Tay–Sach
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::17e80b0fda0ab58b69ef98257471e945
Autor:
William F. Kaemmerer
Publikováno v:
Bioengineering & Translational Medicine
In August 2017, for the first time, a gene therapy was approved for market release in the United States. That approval was followed by two others before the end of the year. This article cites primary literature, review articles concerning particular
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eb1f3b2400fe0b82340eeed2eb5805d4
https://doi.org/10.1002/btm2.10090
https://doi.org/10.1002/btm2.10090
Publikováno v:
Degenerative Neurological and Neuromuscular Disease
Therapies targeting mutant huntingtin DNA, mRNA, and protein have a chance at becoming the first disease-modifying treatments for Huntington’s disease, a fatal inherited neurodegenerative disorder for which only symptom management treatments are av
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::eeb647f9c8e0e07716eba855fa63ee57
https://pubmed.ncbi.nlm.nih.gov/30881191
https://pubmed.ncbi.nlm.nih.gov/30881191
Akademický článek
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Publikováno v:
Optogenetics: From Neuronal Function to Mapping and Disease Biology
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::40f624cbe87e64dba2f6a92f0c9bcf65
https://doi.org/10.1017/9781107281875.014
https://doi.org/10.1017/9781107281875.014
Autor:
Michael D. Kaytor, Zhiming Zhang, Yi Ai, Peter T. Nelson, Richard Grondin, Deepak R. Thakker, William F. Kaemmerer, Marcy R. Weatherspoon, Jennifer M. Heisel, Eric N. Burright, Janelle L. Blum
Publikováno v:
Brain
Huntington's disease is caused by expression of a mutant form of Huntingtin protein containing an expanded polyglutamine repeat. One possible treatment for Huntington's disease may be to reduce expression of mutant Huntingtin in the brain via RNA int
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::66b204c23054f002e86e2c87b2169fb3
https://doi.org/10.1093/brain/awr333
https://doi.org/10.1093/brain/awr333
Autor:
Douglas Ulen Gwost, Verbena Kosovrasti, Michael Meys, Dinah W.Y. Sah, Gregory R. Stewart, Peter A. Hardy, Lubomir Nechev, Pei Ge, Muthiah Manoharan, Klaus Charisse, Yi Ai, Lubomir Tchangov, David K. Stiles, Don M. Gash, Andrei Guzaev, Richard Grondin, Peter T. Nelson, Zhiming Zhang, Brian D. Nelson, William F. Kaemmerer, Martin Maier, Mark T. Butt
Publikováno v:
Experimental Neurology. 233:463-471
Huntington's disease is an autosomal dominant neurodegenerative disease caused by a toxic gain of function mutation in the huntingtin gene (Htt). Silencing of Htt with RNA interference using direct CNS delivery in rodent models of Huntington's diseas
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d4e37e95e67330c02b8b35866ede200a
https://doi.org/10.1016/j.expneurol.2011.11.020
https://doi.org/10.1016/j.expneurol.2011.11.020
Autor:
Matthew D. Johnson, Scott R. Stanslaski, Eric J. Panken, Siddharth Dani, William F. Kaemmerer, Allison T. Connolly, Timothy J. Denison
Publikováno v:
NER
We have found that a set of support vector machines operating upon local field potentials sensed from an implanted DBS lead can identify the contact chosen by the physician for the patient's STN DBS therapy with 91% accuracy. The finding is based on
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::a1199c3e4818114abff2a6fd9f157d4f
https://doi.org/10.1109/ner.2015.7146754
https://doi.org/10.1109/ner.2015.7146754