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In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9
The development of advanced gene and cell therapies for the treatment of genetic diseases requires reliable animal and cellular models to test their efficacy. Moreover, the availability of the target human primary cells of these therapies is reduced in many diseases. The development of endonucleases...
Autores principales: | López-Manzaneda, Sergio, Ojeda-Pérez, Isabel, Zabaleta, Nerea, García-Torralba, Aída, Alberquilla, Omaira, Torres, Raúl, Sánchez-Domínguez, Rebeca, Torella, Laura, Olivier, Emmanuel, Mountford, Joanne, Ramírez, Juan C., Bueren, Juan A., González-Aseguinolaza, Gloria, Segovia, Jose-Carlos |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7683234/ https://www.ncbi.nlm.nih.gov/pubmed/33294491 http://dx.doi.org/10.1016/j.omtm.2020.10.007 |
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