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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Dettagli Bibliografici
Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
Soggetti:
Original Article
Accesso online: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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