未登录 Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis Bainbridge, J. W. B.; Mehat, M. S.; Sundaram, V.; Robbie, S. J.; Barker, S. E.; Ripamonti, C.; Georgiadis, A.; Mowat, F. M.; Beattie, S. G.; Gardner, P. J.; Feathers, K. L.; Luong, V. A.; Yzer, S.; Balaggan, K.; Viswanathan, A.; de Ravel, T. J. L.; Casteels, I.; Holder, G. E.; Tyler, N.; Fitzke, F. W.; Weleber, R. G.; Nardini, M.; Moore, A. T.; Thompson, D. A.; Petersen-Jones, S. M.; Michaelides, M.; van den Born, L. I.; Stockman, A.; Smith, A. J.; Rubin, G.; Ali, R. R. 分享 收藏
NGS library preparation may generate artifactual integration sites of AAV vectors Reply Kaeppel, Christine; Beattie, Stuart G.; Fronza, Raffaele; van Logtenstein, Richard; Salmon, Florence; Schmidt, Sabine; Wolf, Stephan; Nowrouzi, Ali; Glimm, Hanno; von Kalle, Christof; Petry, Harald; Gaudet, Daniel; Schmidt, Manfred 分享 收藏
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates Unzu, Carmen; Hervas-Stubbs, Sandra; Sampedro, Ana; Mauleon, Itsaso; Mancheno, Uxua; Alfaro, Carlos; Enriquez de Salamanca, Rafael; Benito, Alberto; Beattie, Stuart G.; Petry, Harald; Prieto, Jesus; Melero, Ignacio; Fontanellas, Antonio 分享 收藏
TOXICOLOGY AND LIVER TRANSDUCTION EFFICACY EVALUATION OF A RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR IN NON HUMAN PRIMATES AS A POTENTIAL TREATMENT FOR ACUTE INTERMITTENT PORPHYRIA Paneda, A.; Lopez-Franco, E.; Fontanellas, A.; Unzu, C.; Olaguee, C.; Ferrero, R.; Sampedro, A.; Mauleon, I.; Hermening, S.; Beattie, S.; Petry, H.; Ruiz, J.; Benito, A.; Davola, D.; Prieto, J.; Gonzalez-Aseguinolaza, G. 分享 收藏
Development of a Liver-specific Tet-On Inducible System for AAV Vectors and Its Application in the Treatment of Liver Cancer AAV载体肝特异性tet-on诱导系统的研制及其在肝癌治疗中的应用 Vanrell, Lucia; Di Scala, Marianna; Blanco, Laura; Otano, Itziar; Gil-Farina, Irene; Baldim, Victor; Paneda, Astrid; Berraondo, Pedro; Beattie, Stuart G.; Chtarto, Abdelwahed; Tenenbaum, Lilianne; Prieto, Jesus; Gonzalez-Aseguinolaza, Gloria 分享 收藏
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Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice Unzu, Carmen; Sampedro, Ana; Mauleon, Itsaso; Alegre, Manuel; Beattie, Stuart G.; Enriquez de Salamanca, Rafael; Snapper, Jolanda; Twisk, Jaap; Petry, Harald; Gonzalez-Aseguinolaza, Gloria; Artieda, Julio; Rodriguez-Pena, Maria Sol; Prieto, Jesus; Fontanellas, Antonio 分享 收藏
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In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA Cruz, Pedro E.; Mueller, Christian; Cossette, Travis L.; Golant, Alexandra; Tang, Qiushi; Beattie, Stuart G.; Brantly, Mark; Campbell-Thompson, Martha; Blomenkamp, Keith S.; Teckman, Jeffrey H.; Flotte, Terence R. 分享 收藏
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Gene repair validation Graham, IR; Manzano, A; Tagalakis, AD; Mohri, Z; Sperber, G; Hill, V; Beattie, S; Schepelmann, S; Dickson, G; Owen, JS 分享 收藏
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