CRISPR-Cas9 mediated genome editing of Huntington's disease neurospheres

CONCLUSION: Our study confirmed that CAG repeat of R6/2 mouse-derived neurospheres can be edited through CRISPR-Cas9. Editing of CAG repeat sequence decreases polyglutamine aggregation and cellular apoptosis of HD neurospheres, which may be related to the increased expressions of PGC-1α and BDNF. Our data provide the evidence that CRISPR-Cas9 mediated genome editing has therapeutic potential on HD neuronal cells.PMID:36550260 | DOI:10.1007/s11033-022-08175-6
Source: Mol Biol Cell - Category: Molecular Biology Authors: Source Type: research