Work

118. Characterization of Chromosomal Alterations Using a Zinc-Finger Nuclease Targeting the Beta-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

Year: 2016

Type: article

Abstract: The use of engineered nucleases for targeted gene correction of the sickle cell disease (SCD) mutation in hematopoietic stem and progenitor cells (HSPCs) combined with a homologous DNA donor template can result in successful targeted gene correction. However, due to a high sequence homology existing between the beta- and delta-globin genes, off-target endonuclease cleavage events can occur when using endonucleases targeted to beta-globin. Moreover, the introduction of multiple double stranded breaks by site-specific endonucleases has the potential to induce chromosomal alterations such as translocations, deletions, and inversions.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)32927-6