Nuclease-free precise genome editing corrects MECP2 mutations associated with Rett syndrome
Rett syndrome is an acquired progressive neurodevelopmental disorder caused by de novo mutations in the X-linked MECP2 gene which encodes a pleiotropic protein that functions as a global transcriptional regulator and a chromatin modifier. Rett syndrome predominantly affects heterozygous females whil...
Main Authors: | , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Frontiers Media S.A.
2024-03-01
|
Series: | Frontiers in Genome Editing |
Subjects: | |
Online Access: | https://www.frontiersin.org/articles/10.3389/fgeed.2024.1346781/full |