
This innovative CRISPR-based therapy aims to reactivate silenced maternal genes in Prader-Willi Syndrome (PWS) by using dCas9 epigenome editors. The approach targets neuronal cells and has shown promising results in iPSC...
A novel CRISPRi-based therapy utilizing compact Cas9 variants for precise gene silencing of DMPK in myotonic dystrophy, delivered via myo-tropic AAV vectors. This approach aims to reduce disease symptoms and improve pati...