Abstract
Rett syndrome (RTT) is a childhood neurodevelopmental disorder caused by mutations in MECP2. To study the molecular mechanisms underlying RTT, four sublines of H1 hESCs were generated, carrying a hemizygous knockout or mutant allele of MECP2. Exons 3 and 4 of MECP2 were targeted using the CRISPR/Cas9 nuclease system.
Original language | English (US) |
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Journal | Stem Cell Research |
Volume | 40 |
DOIs | |
State | Published - Oct 1 2019 |
Externally published | Yes |