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Precise CCM1 gene correction and inactivation in patient‐derived endothelial cells: Modeling Knudson's two‐hit hypothesis in vitro

BACKGROUND: The CRISPR/Cas9 system has opened new perspectives to study the molecular basis of cerebral cavernous malformations (CCMs) in personalized disease models. However, precise genome editing in endothelial and other hard‐to‐transfect cells remains challenging. METHODS: In a proof‐of‐principl...

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Detalhes bibliográficos
Publicado no:Mol Genet Genomic Med
Main Authors: Spiegler, Stefanie, Rath, Matthias, Much, Christiane D., Sendtner, Barbara S., Felbor, Ute
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6625102/
https://ncbi.nlm.nih.gov/pubmed/31124307
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.755
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