Engineered human Diamond-Blackfan anemia disease model confirms therapeutic effects of clinically applicable lentiviral vector at single-cell resolution

Diamond-Blackfan anemia is a rare genetic bone marrow failure disorder which is usually caused by mutations in ribosomal protein genes. In the present study, we generated a traceable RPS19-deficient cell model using CRISPR-Cas9 and homology-directed repair to investigate the therapeutic effects of a...

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Detalles Bibliográficos
Autores principales: Liu, Yang, Schmiderer, Ludwig, Hjort, Martin, Lang, Stefan, Bremborg, Tyra, Rydström, Anna, Schambach, Axel, Larsson, Jonas, Karlsson, Stefan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Fondazione Ferrata Storti 2023
Materias:
Article - Red Cell Biology & its Disorders
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10620578/
https://www.ncbi.nlm.nih.gov/pubmed/37199130
http://dx.doi.org/10.3324/haematol.2022.282068

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