Ontology highlight
ABSTRACT:
SUBMITTER: Long J
PROVIDER: S-EPMC5835144 | biostudies-literature | 2018 Feb
REPOSITORIES: biostudies-literature
Long Joseph J Hoban Megan D MD Cooper Aaron R AR Kaufman Michael L ML Kuo Caroline Y CY Campo-Fernandez Beatriz B Lumaquin Dianne D Hollis Roger P RP Wang Xiaoyan X Kohn Donald B DB Romero Zulema Z
Molecular therapy : the journal of the American Society of Gene Therapy 20171109 2
The use of engineered nucleases combined with a homologous DNA donor template can result in targeted gene correction of the sickle cell disease mutation in hematopoietic stem and progenitor cells. However, because of the high homology between the adjacent human β- and δ-globin genes, off-target cleavage is observed at δ-globin when using some endonucleases targeted to the sickle mutation in β-globin. Introduction of multiple double-stranded breaks by endonucleases has the potential to induce int ...[more]