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...

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Dades bibliogràfiques
Publicat a:Mol Ther Methods Clin Dev
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
Matèries:
Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7683234/
https://ncbi.nlm.nih.gov/pubmed/33294491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.10.007
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