Genomics

Dataset Information

118

Homo sapiens


ABSTRACT: Targeted sequencing of potential CRISRP/Cas9 off-target editing sites in modified human CD34+ Haematopoietic Stem Cells

ORGANISM(S): Homo sapiens  

PROVIDER: PRJNA376778 | ENA |

REPOSITORIES: ENA

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Publications


β-Thalassemia is one of the most common inherited anemias, with no effective cure for most patients. The pathophysiology reflects an imbalance between α- and β-globin chains with an excess of free α-globin chains causing ineffective erythropoiesis and hemolysis. When α-thalassemia is co-inherited with β-thalassemia, excess free α-globin chains are reduced significantly ameliorating the clinical severity. Here we demonstrate the use of CRISPR/Cas9 genome editing of primary human hematopoietic ste  ...[more]

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