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Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type NF1外显子17的靶向外显子跳跃治疗神经纤维瘤病类型 Leier, Andre; Moore, Marc; Liu, Hui; Daniel, Michael; Hyde, Alexis M.; Messiaen, Ludwine; Korf, Bruce R.; Selvakumaran, Jamuna; Ciszewski, Lukasz; Lambert, Laura; Foote, Jeremy; Wallace, Margaret R.; Kesterson, Robert A.; Dickson, George; Popplewell, Linda; Wallis, Deeann 分享 收藏
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BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy Strings-Ufombah, Vanessa; Malerba, Alberto; Kao, Shih-Chu; Harbaran, Sonal; Roth, Fanny; Cappellari, Ornella; Lu-Nguyen, Ngoc; Takahashi, Keiko; Mukadam, Sophie; Kilfoil, Georgina; Kloth, Claudia; Roelvink, Petrus; Dickson, George; Trollet, Capucine; Suhy, David 分享 收藏
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RAC1B modulates intestinal tumourigenesis via modulation of WNT and EGFR signalling pathways Gudino, Victoria; Pohl, Sebastian Other-Gee; Billard, Caroline V.; Cammareri, Patrizia; Bolado, Alfonso; Aitken, Stuart; Stevenson, David; Hall, Adam E.; Agostino, Mark; Cassidy, John; Nixon, Colin; von Kriegsheim, Alex; Freile, Paz; Popplewell, Linda; Dickson, George; Murphy, Laura; Wheeler, Ann; Dunlop, Malcolm; Din, Farhat; Strathdee, Douglas; Sansom, Owen J.; Myant, Kevin B. 分享 收藏
Mutation-Directed Therapeutics for Neurofibromatosis Type I Leier, Andre; Bedwell, David M.; Chen, Ann T.; Dickson, George; Keeling, Kim M.; Kesterson, Robert A.; Korf, Bruce R.; Lago, Tatiana T. Marquez; Mueller, Ulrich F.; Popplewell, Linda; Zhou, Jiangbing; Wallis, Deeann 分享 收藏
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy Frank, Diane E.; Schnell, Frederick J.; Akana, Cody; El-Husayni, Saleh H.; Desjardins, Cody A.; Morgan, Jennifer; Charleston, Jay S.; Sardone, Valentina; Domingos, Joana; Dickson, George; Straub, Volker; Guglieri, Michela; Mercuri, Eugenio; Servais, Laurent; Muntoni, Francesco 分享 收藏
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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy 肌肉生长抑制素抑制联合反义寡核苷酸治疗可改善脊髓性肌萎缩症的预后 Zhou, Haiyan; Meng, Jinhong; Malerba, Alberto; Catapano, Francesco; Sintusek, Palittiya; Jarmin, Susan; Feng, Lucy; Ngoc Lu-Nguyen; Sun, Lianwen; Mariot, Virginie; Dumonceaux, Julie; Morgan, Jennifer E.; Gissen, Paul; Dickson, George; Muntoni, Francesco 分享 收藏
Vaccination of Macaques with DNA Followed by Adenoviral Vectors Encoding Simian Immunodeficiency Virus (SIV) Gag Alone Delays Infection by Repeated Mucosal Challenge with SIV Almond, Neil; Berry, Neil; Stebbings, Richard; Preston, Mark; Ham, Claire; Page, Mark; Ferguson, Debbie; Rose, Nicola; Li, Bo; Mee, Edward T.; Hassall, Mark; Stahl-Hennig, Christiane; Athanasopoulos, Takis; Papagatsias, Timos; Herath, Shanthi; Benlahrech, Adel; Dickson, George; Meiser, Andrea; Patterson, Steven 分享 收藏
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Next-generation muscle-directed gene therapy by in silico vector design Sarcar, S.; Tulalamba, W.; Rincon, M. Y.; Tipanee, J.; Pham, H. Q.; Evens, H.; Boon, D.; Samara-Kuko, E.; Keyaerts, M.; Loperfido, M.; Berardi, E.; Jarmin, S.; In't Veld, P.; Dickson, G.; Lahoutte, T.; Sampaolesi, M.; De Bleser, P.; VandenDriessche, T.; Chuah, M. K. 分享 收藏
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy Malerba, Alberto; Roth, Fanny; Harish, Pradeep; Dhiab, Jamila; Ngoc Lu-Nguyen; Cappellari, Ornella; Jarmin, Susan; Mahoudeau, Alexandrine; Ythier, Victor; Laine, Jeanne; Negroni, Elisa; Abgueguen, Emmanuelle; Simonelig, Martine; Guedat, Philippe; Mouly, Vincent; Butler-Browne, Gillian; Voisset, Cecile; Dickson, George; Trollet, Capucine 分享 收藏
Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy Benedetti, Sara; Uno, Narumi; Hoshiya, Hidetoshi; Ragazzi, Martina; Ferrari, Giulia; Kazuki, Yasuhiro; Moyle, Louise Anne; Tonlorenzi, Rossana; Lombardo, Angelo; Chaouch, Soraya; Mouly, Vincent; Moore, Marc; Popplewell, Linda; Kazuki, Kanako; Katoh, Motonobu; Naldini, Luigi; Dickson, George; Messina, Graziella; Oshimura, Mitsuo; Cossu, Giulio; Tedesco, Francesco Saverio 分享 收藏
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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy Le Guiner, Caroline; Servais, Laurent; Montus, Marie; Larcher, Thibaut; Fraysse, Bodvael; Moullec, Sophie; Allais, Marine; Francois, Virginie; Dutilleul, Maeva; Malerba, Alberto; Koo, Taeyoung; Thibaut, Jean-Laurent; Matot, Beatrice; Devaux, Marie; Le Duff, Johanne; Deschamps, Jack-Yves; Barthelemy, Ines; Blot, Stephane; Testault, Isabelle; Wahbi, Karim; Ederhy, Stephane; Martin, Samia; Veron, Philippe; Georger, Christophe; Athanasopoulos, Takis; Masurier, Carole; Mingozzi, Federico; Carlier, Pierre; Gjata, Bernard; Hogrel, Jean-Yves; Adjali, Oumeya; Mavilio, Fulvio; Voit, Thomas; Moullier, Philippe; Dickson, George 分享 收藏
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