Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β(0)39-thalassemia patients

Gene editing by the CRISPR-Cas9 nuclease system technology can be considered among the most promising strategies to correct hereditary mutations in a variety of monogenic diseases. In this paper, we present for the first time the correction, by CRISPR-Cas9 gene editing, of the β(0)39-thalassemia mut...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Cosenza, Lucia Carmela, Gasparello, Jessica, Romanini, Nicola, Zurlo, Matteo, Zuccato, Cristina, Gambari, Roberto, Finotti, Alessia
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8091488/
https://ncbi.nlm.nih.gov/pubmed/33997100
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.03.025
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