High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot
Susanna Croci, Miriam Lucia Carriero, Katia Capitani, Sergio Daga, Francesco Donati, Elisa Frullanti, Vittoria Lamacchia, Rossella Tita, Annarita Giliberti, Floriana Valentino, Elisa Benetti, Annalisa Ciabattini, Simone Furini, Caterina Lo Rizzo, Anna Maria Pinto, Silvestro G. Conticello, Alessandra Renieri, Ilaria Meloni
Topics & Concepts
Rett syndromeMECP2BiologyGenome editingInduced pluripotent stem cellCRISPRReprogrammingCas9GeneGeneticsIndelGenetic enhancementComputational biologyBioinformaticsEmbryonic stem cellPhenotypeSingle-nucleotide polymorphismGenotypeCRISPR and Genetic EngineeringGenetics and Neurodevelopmental DisordersRNA Interference and Gene Delivery