NeuroscienceFrontiers in Molecular NeurosciencePeer-reviewed
Targeted Reactivation of FMR1 Transcription in Fragile X Syndrome Embryonic Stem Cells
Jill M. Haenfler, Geena Skariah, Caitlin M. Rodriguez…·Peter K. Todd
This study reactivates FMR1 transcription in FXS patient cells using CRISPR/dCAS9 and VP192, increasing FMRP levels and demonstrating a potential therapeutic approach for Fragile X Syndrome.
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