Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients

Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-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 β039-thalassemia mutat...

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Bibliographic Details
Main Authors: Lucia Carmela Cosenza, Jessica Gasparello, Nicola Romanini, Matteo Zurlo, Cristina Zuccato, Roberto Gambari, Alessia Finotti
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
CRISPR-Cas9
genome editing
β039-thalassemia
erythroid precursors
β-globin
personalized therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000656

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http://www.sciencedirect.com/science/article/pii/S2329050121000656

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