Result Number | Material Type | Add to My Shelf Action | Record Details and Options |
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1 |
Material Type: Artigo
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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndromeMagnani, A ; Semeraro, M ; Adam, F ; Booth, C ; Dupré, L ; Morris, E C ; Gabrion, A ; Roudaut, C ; Borgel, D ; Toubert, A ; Clave, E ; Abdo, C ; Gorochov, G ; Petermann, R ; Guiot, M ; Miyara, M ; Moshous, D ; Magrin, E ; Denis, A ; Suarez, F ; Lagresle, C ; Roche, A M ; Everett, J ; Trinquand, A ; Guisset, M ; Bayford, J Xu ; Hacein-Bey-Abina, S ; Kauskot, A ; Elfeky, R ; Rivat, C ; Abbas, S ; Gaspar, H B ; Macintyre, E ; Picard, C ; Bushman, F D ; Galy, A ; Fischer, A ; Six, E ; Thrasher, A J ; Cavazzana, MNature Medicine, 2022-01, Vol.28 (1), p.71-80 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |
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2 |
Material Type: Artigo
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Induced pluripotent stem cells in disease modelling and drug discoveryRowe, R Grant ; Daley, George QNature reviews. Genetics, 2019-07, Vol.20 (7), p.377-388 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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3 |
Material Type: Artigo
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MicroRNA therapeutics: towards a new era for the management of cancer and other diseasesRupaimoole, Rajesha ; Slack, Frank JNature reviews. Drug discovery, 2017-03, Vol.16 (3), p.203-222 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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4 |
Material Type: Artigo
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Delivery technologies for genome editingYin, Hao ; Kauffman, Kevin J ; Anderson, Daniel GNature reviews. Drug discovery, 2017-06, Vol.16 (6), p.387-399 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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5 |
Material Type: Artigo
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Clinical use of lentiviral vectorsMilone, Michael C ; O'Doherty, UnaLeukemia, 2018-07, Vol.32 (7), p.1529-1541 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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6 |
Material Type: Artigo
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Engineering adeno-associated viruses for clinical gene therapyKotterman, Melissa A ; Schaffer, David VNature reviews. Genetics, 2014-07, Vol.15 (7), p.445-451 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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7 |
Material Type: Artigo
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The clinical landscape for AAV gene therapiesKuzmin, Dmitry A ; Shutova, Maria V ; Johnston, Natalie R ; Smith, Owen P ; Fedorin, Vasily V ; Kukushkin, Yury S ; van der Loo, Johannes C M ; Johnstone, Elaine CNature reviews. Drug discovery, 2021-03, Vol.20 (3), p.173-174 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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8 |
Material Type: Artigo
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Noncoding RNA therapeutics - challenges and potential solutionsWinkle, Melanie ; El-Daly, Sherien M ; Fabbri, Muller ; Calin, George ANature reviews. Drug discovery, 2021-08, Vol.20 (8), p.629-651 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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9 |
Material Type: Artigo
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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integrationSuzuki, Keiichiro ; Tsunekawa, Yuji ; Hernandez-Benitez, Reyna ; Wu, Jun ; Zhu, Jie ; Kim, Euiseok J ; Hatanaka, Fumiyuki ; Yamamoto, Mako ; Araoka, Toshikazu ; Li, Zhe ; Kurita, Masakazu ; Hishida, Tomoaki ; Li, Mo ; Aizawa, Emi ; Guo, Shicheng ; Chen, Song ; Goebl, April ; Soligalla, Rupa Devi ; Qu, Jing ; Jiang, Tingshuai ; Fu, Xin ; Jafari, Maryam ; Esteban, Concepcion Rodriguez ; Berggren, W Travis ; Lajara, Jeronimo ; Nuñez-Delicado, Estrella ; Guillen, Pedro ; Campistol, Josep M ; Matsuzaki, Fumio ; Liu, Guang-Hui ; Magistretti, Pierre ; Zhang, Kun ; Callaway, Edward M ; Zhang, Kang ; Belmonte, Juan Carlos IzpisuaNature (London), 2016-12, Vol.540 (7631), p.144-149 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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10 |
Material Type: Artigo
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Treatment of a metabolic liver disease by in vivo genome base editing in adult miceVilliger, Lukas ; Grisch-Chan, Hiu Man ; Lindsay, Helen ; Ringnalda, Femke ; Pogliano, Chiara B ; Allegri, Gabriella ; Fingerhut, Ralph ; Häberle, Johannes ; Matos, Joao ; Robinson, Mark D ; Thöny, Beat ; Schwank, GeraldNature medicine, 2018-10, Vol.24 (10), p.1519-1525 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |