Zobrazeno 1 - 10
of 439
pro vyhledávání: '"K. Kaspar"'
Autor:
Tyler B. Johnson, Jon J. Brudvig, Shibi Likhite, Melissa A. Pratt, Katherine A. White, Jacob T. Cain, Clarissa D. Booth, Derek J. Timm, Samantha S. Davis, Brandon Meyerink, Ricardo Pineda, Cassandra Dennys-Rivers, Brian K. Kaspar, Kathrin Meyer, Jill M. Weimer
Publikováno v:
Frontiers in Genetics, Vol 14 (2023)
CLN3 disease, caused by biallelic mutations in the CLN3 gene, is a rare pediatric neurodegenerative disease that has no cure or disease modifying treatment. The development of effective treatments has been hindered by a lack of etiological knowledge,
Externí odkaz:
https://doaj.org/article/cb379df157064165b746745383d34428
Autor:
Katherine A. White, Hemanth R. Nelvagal, Timothy A. Poole, Bin Lu, Tyler B. Johnson, Samantha Davis, Melissa A. Pratt, Jon Brudvig, Ana B. Assis, Shibi Likhite, Kathrin Meyer, Brian K. Kaspar, Jonathan D. Cooper, Shaomei Wang, Jill M. Weimer
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 497-507 (2021)
Batten disease is a family of rare, fatal, neuropediatric diseases presenting with memory/learning decline, blindness, and loss of motor function. Recently, we reported the use of an AAV9-mediated gene therapy that prevents disease progression in a m
Externí odkaz:
https://doaj.org/article/dc4e787451044bffb7592fb3419f7757
Autor:
Cátia Gomes, Catarina Sequeira, Shibi Likhite, Cassandra N. Dennys, Stephen J. Kolb, Pamela J. Shaw, Ana R. Vaz, Brian K. Kaspar, Kathrin Meyer, Dora Brites
Publikováno v:
Cells, Vol 11, Iss 7, p 1186 (2022)
A lack of stratification methods in patients with amyotrophic lateral sclerosis (ALS) is likely implicated in therapeutic failures. Regional diversities and pathophysiological abnormalities in astrocytes from mice with SOD1 mutations (mSOD1-ALS) can
Externí odkaz:
https://doaj.org/article/b22c77976eb9481a93281f8a06e61364
Autor:
Tommaso Iannitti, Joseph M. Scarrott, Shibi Likhite, Ian R.P. Coldicott, Katherine E. Lewis, Paul R. Heath, Adrian Higginbottom, Monika A. Myszczynska, Marta Milo, Guillaume M. Hautbergue, Kathrin Meyer, Brian K. Kaspar, Laura Ferraiuolo, Pamela J. Shaw, Mimoun Azzouz
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 75-88 (2018)
Of familial amyotrophic lateral sclerosis (fALS) cases, 20% are caused by mutations in the gene encoding human cytosolic Cu/Zn superoxide dismutase (hSOD1). Efficient translation of the therapeutic potential of RNAi for the treatment of SOD1-ALS pati
Externí odkaz:
https://doaj.org/article/b30506436b9849bf842271b7ed602925
Autor:
Guillaume M. Hautbergue, Lydia M. Castelli, Laura Ferraiuolo, Alvaro Sanchez-Martinez, Johnathan Cooper-Knock, Adrian Higginbottom, Ya-Hui Lin, Claudia S. Bauer, Jennifer E. Dodd, Monika A. Myszczynska, Sarah M. Alam, Pierre Garneret, Jayanth S. Chandran, Evangelia Karyka, Matthew J. Stopford, Emma F. Smith, Janine Kirby, Kathrin Meyer, Brian K. Kaspar, Adrian M. Isaacs, Sherif F. El-Khamisy, Kurt J. De Vos, Ke Ning, Mimoun Azzouz, Alexander J. Whitworth, Pamela J. Shaw
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-18 (2017)
The RNA for ALS- and frontotemporal dementia-associated C9ORF72 gene is exported from nucleus via an unknown mechanism. This study shows that reduction of nuclear export adaptor SRSF1 can alleviate neuronal cell death and nuclear export of C9ORF72 in
Externí odkaz:
https://doaj.org/article/26d01605fb7344faaa67f88fe2c80e8f
Autor:
Rebecca Brulet, Taito Matsuda, Ling Zhang, Carlos Miranda, Mauro Giacca, Brian K. Kaspar, Kinichi Nakashima, Jenny Hsieh
Publikováno v:
Stem Cell Reports, Vol 8, Iss 6, Pp 1506-1515 (2017)
Currently, all methods for converting non-neuronal cells into neurons involve injury to the brain; however, whether neuronal transdifferentiation can occur long after the period of insult remains largely unknown. Here, we use the transcription factor
Externí odkaz:
https://doaj.org/article/d22afb285af14f2283106a4825715274
Akademický článek
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Akademický článek
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Autor:
Marybeth Camboni, Chiou-Miin Wang, Carlos Miranda, Jung Hae Yoon, Rui Xu, Deborah Zygmunt, Brian K. Kaspar, Paul T. Martin
Publikováno v:
Neurobiology of Disease, Vol 62, Iss , Pp 31-43 (2014)
Recent clinical and pre-clinical studies suggest that both active and passive immunization strategies targeting Aβ amyloid may have clinical benefit in Alzheimer's disease. Here, we demonstrate that vaccination of APPswePSEN1dE9 mice with SDPM1, an
Externí odkaz:
https://doaj.org/article/ea58be5f665549689ab0cfe161b984cc
Autor:
Atsushi Miyanohara, Kota Kamizato, Stefan Juhas, Jana Juhasova, Michael Navarro, Silvia Marsala, Nada Lukacova, Marian Hruska-Plochan, Erik Curtis, Brandon Gabel, Joseph Ciacci, Eric T Ahrens, Brian K Kaspar, Don Cleveland, Martin Marsala
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Effective in vivo use of adeno-associated virus (AAV)-based vectors to achieve gene-specific silencing or upregulation in the central nervous system has been limited by the inability to provide more than limited deep parenchymal expression in adult a
Externí odkaz:
https://doaj.org/article/2656793c9c4b4a1bb3e8f01a16e8dd29