Publication TitleJournalAuthorsPub DatePubMedProject PIs
Identification of a Guide RNA Targeting an Ultraconserved Element for Evaluation of Cas9 Genome Editors Across Mammalian SpeciesThe CRISPR JournalGowen et al. 9/23/2439308435Saha
Regulatory Considerations for Genome-Edited T-cell TherapiesCancer Immunology ResearchJadlowsky et al. 9/3/2439018097Spencer, Duncavage
Animal and bacterial viruses share conserved mechanisms of immune evasionCellHobbs et al. 8/27/2439197447Doudna
Birth of protein folds and functions in the viromeNatureNomburg et al. 8/26/2439187718Doudna
Motion blur microscopy: in vitro imaging of cell adhesion dynamics in whole blood flowNature CommunicationsGoreke et al. 8/16/2439152149Giannikopoulos, Gurkan
Zfp106 binds to G-quadruplex RNAs and inhibits RAN translation and formation of RNA foci caused by G4C2 repeatsProceedings of the National Academy of Sciences of the United States of AmericaCelona et al. 7/23/2439042693Doudna
Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editorScienceNeumann et al. 6/28/2438935715Vallabh
Interferon-γ induces combined pyroptotic angiopathy and APOL1 expression in human kidney diseaseCell ReportsJuliar et al. 6/25/2438838223Freedman
Rapid DNA unwinding accelerates genome editing by engineered CRISPR-Cas9CellEggers et al. 5/22/2438781968Doudna
Selective haematological cancer eradication with preserved haematopoiesisNatureGaraude et al. 5/22/2438778101Tsai
An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene deliveryScienceHuang et al. 5/16/2438753766Vallabh
Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibilityStem cell reportsPamies et al. 5/14/2438670111Freedman
Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in miceThe Journal of Clinical InvestigationRiley et al. 5/9/2438950310Peranteau, Musunuru
Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shiftThe AnalystSekyonda et al. 4/29/2438501195Giannikopoulos, Gurkan
Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excisionProceedings of the National Academy of Sciences of the United States of AmericaSachdev et al. 4/15/2438621131Doudna
Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney diseaseCell Stem CellVishy et al. 4/4/2438579684Freedman
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophyNature Biomedical Engineering Alves et al.3/8/2438057426Tsai
Building CRISPR Gene Therapies for the Central Nervous System: A ReviewJAMA NeurologySalomonsson & Clelland3/1/2438285472Doudna
Engineering self-deliverable ribonucleoproteins for genome editing in the brainNature CommunicationsChen et al. 2/26/2438409124Doudna
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
Validated assays for the quantification of C9orf72 human pathologyScientific ReportsSalomonsson et al.1/8/2438191789Doudna
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