Article

Development of Gene Editing Strategies for Human <b>β</b> -Globin (HBB) Gene Mutations

2020-01-17

Abstract excerpt

Recent developments in gene editing technology have enabled scientists to modify DNA sequence by using engineered endonucleases. These gene editing tools are promising candidates for clinical applications, especially for treatment of inherited disorders like sickle cell disease (SCD). SCD is caused by a point mutation in human β-globin gene (HBB). Clinical strategies have demonstrated substantial success, however...

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Literature Corpus work: cb922e93-2090-512f-afc0-b361ebba9255
DOI: 10.1101/2020.01.16.908319

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