Publication TitleJournalAuthorsPub DatePubMedProject PIs
Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney diseaseCell Stem CellVishy et al. 4/4/2438579684Freedman
Validated assays for the quantification of C9orf72 human pathologyScientific ReportsSalomonsson et al.1/8/2438191789Doudna
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophyNature Biomedical Engineering Alves et al.3/8/2438057426Tsi
Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preferenceNature CommunicationsZhang et al.2/24/2438402281Lutz, Arbab, Grey, Liu, Mouro Pinto
Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical needAmerican Journal of Human GeneticsBrooks et al.11/3/2337924808Peranteau, Musunuru
A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variantHGG AdvancesBrooks et al.10/31/2337922902Peranteau, Musunuru
Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral InjectionBiochemistryTuma et al.9/20/2337729550Doudna | Bankiewicz, Murthy
Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editingNature CommunicationsBrooks et al.6/10/2337301931Peranteau, Musunuru