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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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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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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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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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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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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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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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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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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 |
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Material Type: Artigo
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Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancerLu, You ; Xue, Jianxin ; Deng, Tao ; Zhou, Xiaojuan ; Yu, Kun ; Deng, Lei ; Huang, Meijuan ; Yi, Xin ; Liang, Maozhi ; Wang, Yu ; Shen, Haige ; Tong, Ruizhan ; Wang, Wenbo ; Li, Li ; Song, Jin ; Li, Jing ; Su, Xiaoxing ; Ding, Zhenyu ; Gong, Youling ; Zhu, Jiang ; Wang, Yongsheng ; Zou, Bingwen ; Zhang, Yan ; Li, Yanying ; Zhou, Lin ; Liu, Yongmei ; Yu, Min ; Wang, Yuqi ; Zhang, Xuanwei ; Yin, Limei ; Xia, Xuefeng ; Zeng, Yong ; Zhou, Qiao ; Ying, Binwu ; Chen, Chong ; Wei, Yuquan ; Li, Weimin ; Mok, TonyNature medicine, 2020-05, Vol.26 (5), p.732-740 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |