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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...

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Enregistré dans:
Détails bibliographiques
Publié dans:Mol Ther Methods Clin Dev
Auteurs principaux: 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
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2020
Sujets:
Accès en ligne: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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