Zobrazeno 1 - 10
of 318
pro vyhledávání: '"George, Dickson"'
Autor:
André Leier, Marc Moore, Hui Liu, Michael Daniel, Alexis M. Hyde, Ludwine Messiaen, Bruce R. Korf, Jamuna Selvakumaran, Lukasz Ciszewski, Laura Lambert, Jeremy Foote, Margaret R. Wallace, Robert A. Kesterson, George Dickson, Linda Popplewell, Deeann Wallis
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 28, Iss , Pp 261-278 (2022)
We investigated the feasibility of utilizing an exon-skipping approach as a genotype-dependent therapeutic for neurofibromatosis type 1 (NF1) by determining which NF1 exons might be skipped while maintaining neurofibromin protein expression and GTPas
Externí odkaz:
https://doaj.org/article/e537f0d08c7e4a27859b8efc3e84fa46
Autor:
Vanessa Strings-Ufombah, Alberto Malerba, Shih-Chu Kao, Sonal Harbaran, Fanny Roth, Ornella Cappellari, Ngoc Lu-Nguyen, Keiko Takahashi, Sophie Mukadam, Georgina Kilfoil, Claudia Kloth, Petrus Roelvink, George Dickson, Capucine Trollet, David Suhy
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 24, Iss , Pp 67-78 (2021)
Oculopharyngeal muscular dystrophy (OPMD) is a rare autosomal dominant disease that results from an alanine expansion in the N-terminal domain of Poly-A Binding Protein Nuclear-1 (PABPN1). We have recently demonstrated that a two-vector gene therapy
Externí odkaz:
https://doaj.org/article/a38e66f16de34830a7d74e6ea8d9e973
Autor:
Victoria Gudiño, Sebastian Öther-Gee Pohl, Caroline V. Billard, Patrizia Cammareri, Alfonso Bolado, Stuart Aitken, David Stevenson, Adam E. Hall, Mark Agostino, John Cassidy, Colin Nixon, Alex von Kriegsheim, Paz Freile, Linda Popplewell, George Dickson, Laura Murphy, Ann Wheeler, Malcolm Dunlop, Farhat Din, Douglas Strathdee, Owen J. Sansom, Kevin B. Myant
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
RAC1 is a downstream target of the Wnt signaling that promotes intestinal stem cell expansion and tumorigenesis. Here, the authors identify the specific splice variant RAC1B as an important mediator of colorectal tumourigenesis and a potential target
Externí odkaz:
https://doaj.org/article/0d5fa07d90ab4bcca894bd613a859024
Autor:
Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni
Publikováno v:
Journal of Cachexia, Sarcopenia and Muscle, Vol 11, Iss 3, Pp 768-782 (2020)
Abstract Background Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1) gene that lead to SMN deficiency. Different SMN‐restoring therapies substantially prolong survival and function in transgenic mice
Externí odkaz:
https://doaj.org/article/833a799c692e44558f9719c738478c3a
Autor:
Andre Leier, David M. Bedwell, Ann T. Chen, George Dickson, Kim M. Keeling, Robert A. Kesterson, Bruce R. Korf, Tatiana T. Marquez Lago, Ulrich F. Müller, Linda Popplewell, Jiangbing Zhou, Deeann Wallis
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 20, Iss , Pp 739-753 (2020)
Significant advances in biotechnology have led to the development of a number of different mutation-directed therapies. Some of these techniques have matured to a level that has allowed testing in clinical trials, but few have made it to approval by
Externí odkaz:
https://doaj.org/article/2f47ed29af8d44b7896912a5345ddaf9
Autor:
Pradeep Harish, Alberto Malerba, Ngoc Lu‐Nguyen, Leysa Forrest, Ornella Cappellari, Fanny Roth, Capucine Trollet, Linda Popplewell, George Dickson
Publikováno v:
Journal of Cachexia, Sarcopenia and Muscle, Vol 10, Iss 5, Pp 1016-1026 (2019)
Abstract Background Oculopharyngeal muscular dystrophy (OPMD) is a late‐onset muscle disease affecting one per 80 000 of the general population characterized by profound dysphagia and ptosis, and limb weakness at later stages. Affected muscles are
Externí odkaz:
https://doaj.org/article/c5412e71a06841f79b1919d787c6f2b6
Publikováno v:
Biomedicines, Vol 10, Iss 7, p 1623 (2022)
Silencing the expression of the double homeobox 4 (DUX4) gene offers great potential for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Several research groups have recently reported promising results using systemic antisense therapy
Externí odkaz:
https://doaj.org/article/33aa5b736f17432e9260886883792b79
Autor:
Pradeep Harish, Leysa Forrest, Shanti Herath, George Dickson, Alberto Malerba, Linda Popplewell
Publikováno v:
Frontiers in Physiology, Vol 11 (2020)
BackgroundOculopharyngeal muscular dystrophy (OPMD) is a late-onset muscle disease presented by ptosis, dysphagia, and limb weakness. Affected muscles display increased fibrosis and atrophy, with characteristic inclusion bodies in the nucleus. Myosta
Externí odkaz:
https://doaj.org/article/02a3c9eeba49442996a256803a59c437
Autor:
Sara Benedetti, Narumi Uno, Hidetoshi Hoshiya, Martina Ragazzi, Giulia Ferrari, Yasuhiro Kazuki, Louise Anne Moyle, Rossana Tonlorenzi, Angelo Lombardo, Soraya Chaouch, Vincent Mouly, Marc Moore, Linda Popplewell, Kanako Kazuki, Motonobu Katoh, Luigi Naldini, George Dickson, Graziella Messina, Mitsuo Oshimura, Giulio Cossu, Francesco Saverio Tedesco
Publikováno v:
EMBO Molecular Medicine, Vol 10, Iss 2, Pp 254-275 (2017)
Abstract Transferring large or multiple genes into primary human stem/progenitor cells is challenged by restrictions in vector capacity, and this hurdle limits the success of gene therapy. A paradigm is Duchenne muscular dystrophy (DMD), an incurable
Externí odkaz:
https://doaj.org/article/16f94df9ebc24f2b8c6332a79a02b829
Publikováno v:
Neurobiology of Disease, Vol 106, Iss , Pp 63-75 (2017)
Alzheimer's disease is the most prevalent form of dementia in the elderly but the precise causal mechanisms are still not fully understood. Growing evidence supports a significant role for Aβ42 oligomers in the development and progression of Alzheim
Externí odkaz:
https://doaj.org/article/00763ccb102541e8a52a93771125d2e4