Publication TitleJournalAuthorsPub DatePubMedProject PIs
Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transferMolecular therapySmith et al. 7/3/2438715362Tarantal, Hartigan-O'Connor, Segal
Bioengineered Model of Human LGMD2B Skeletal Muscle Reveals Roles of Intracellular Calcium Overload in Contractile and Metabolic Dysfunction in DysferlinopathyAdvanced scienceKhodabukus et al.6/17/2438887849Gersbach, Bursac, Truskey
Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editingNature Biomedical EngineeringPandey et al. 6/10/2438858586Liu
Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapiesMolecular TherapySkeate el a. 6/5/2438627969Carlson
AAV5 delivery of CRISPR/Cas9 mediates genome editing in the lungs of young rhesus monkeysHuman Gene TherapyXue et al. 5/20/2438767512Gao, Anderson, Xue | Tarantal
Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoysNature CommunicationsPiotrowski-Daspit et al. 5/18/2438762483Saltzman, Glazer
Modifying the Backbone Chemistry of PEG-Based Bottlebrush Block Copolymers for the Formation of Long-Circulating NanoparticlesAdvanced healthcare MaterialsGrundler et al. 5/11/2438734871Glazer, Ly, Saltzman
A novel humanized mouse model for HIV and tuberculosis co-infection studiesFrontiers in ImmunologyBohorquez et al. 5/10/2438799434Yi
Imaging CAR-NK cells targeted to HER2 ovarian cancer with human sodium-iodide symporter-based positron emission tomographyEuropean Journal of Nuclear Medicine and Molecular ImagingShalaby et al.5/9/2438722382Ronald
Imaging of adeno-associated viral capsids for purposes of gene editing using CEST NMR/MRIMagnetic Resonance in MedicineLam et al. 4/23/2438651648Vandsburger
Adeno-associated virus as a delivery vector for gene therapy of human diseasesSignal Transduction and Targeted TherapyWang et al.4/3/2438565561Gao, Anderson, Xue
High-throughput evaluation of genetic variants with prime editing sensor librariesNature BiotechnologyGould et al. 3/12/2438472508Liu
Bacteriophage therapy for the treatment of Mycobacterium tuberculosis infections in humanized miceCommunications BiologyYang et al. 3/9/2438461214Yi
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
Targeted nonviral delivery of genome editors in vivoProceedings of the National Academy of SciencesTsuchida et al.3/4/2438437567Doudna, Banfield
Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heartProceedings of the National Academy of SciencesRadmand et al. 3/4/2438437539Dahlman, Santangelo
A humanized mouse model for adeno-associated viral gene therapyNature CommunicationsBarzi et al. 3/4/2438438373Gersbach, Bursac, Truskey
Compartmentalized ocular lymphatic system mediates eye-brain immunityNatureYin et al. 2/28/2438418880Saltzman, Glazer
Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preferenceNature CommunicationsZhang et al.2/24/2438402281Liu
Advancements in therapeutic development: kidney organoids and organs on a chipKidney InternationalTabibzadeh and Morizane1/29/2438296026Morizane, Lewis, Sabbisetti
CRISPR Activation Reverses Haploinsufficiency and Functional Deficits Caused by TTN Truncation VariantsCirculationGhahremani et al. 1/18/2438235591Hinson
In vivo human T cell engineering with enveloped delivery vehiclesNature BiotechnologyHamilton et al.1/11/2438212493Doudna, Banfield
Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cellsProceedings of the National Academy of SciencesGao et al.1/9/2438150477Chen, Liu, Xu
Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivoNature BiotechnologyAn et al.1/8/2438191664Liu
Protein-based nanoparticles for therapeutic nucleic acid deliveryBiomaterialsEweje et al. 1/2/2438181574Chaikof
Tumor-tropic Trojan horses: Using mesenchymal stem cells as cellular nanotheranosticsTheranosticsRosu et al. 1/1/2438169524Bulte
AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfectaMolecular Therapy Nucleic AcidsYang et al.12/26/2338261950Gao, Anderson, Xue
Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymesNucleic Acid ResearchYoon et al.12/11/2337971304Doudna, Banfield
Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivoNature CommunicationsKulhankova et al.12/5/2338052872McCray | Liu | Tarantal
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivoNucleic Acid ResearchZhang et al.11/30/2338033325Sontheimer, Khvorova, Watts, Wolfe | Liu
Combinatorial development of nebulized mRNA delivery formulations for the lungsNature NanotechnologyJiang et al.11/20/2337985700Gao, Anderson, Xue
Assigning functionality to cysteines by base editing of cancer dependency genesNature Chemical BiologyLi et al.11/19/2337783940Liu
Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screensNature GeneticsMcCutcheon et al.11/9/2337945901Gersbach
CasPEDIA Database: a functional classification system for class 2 CRISPR-Cas enzymesNucleic Acid ResearchAdler et al.10/27/2337889041Doudna, Banfield
DNA recognition and induced genomemodification by a hydroxymethyl-g tail-clamppeptide nucleic acidCell Reports Physical ScienceOyaghire et al. 10/18/2337920723Glazer, Ly, Saltzman
Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cellsCellTsuchida et al.10/12/2337794590Doudna, Banfield
Structure-guided AAV capsid evolution strategies for enhanced CNS gene deliveryNature ProtocolsGonzalez et al.9/21/2337735235Asokan
Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral InjectionBiochemistryTuma et al.9/20/2337729550Doudna | Bankiewicz, Murthy
Targeted genome editing with a DNA-dependent DNA polymerase and exogenous DNA-containing templatesNature BiotechnologyLiu et al.9/14/2337709915Sontheimer, Khvorova, Watts, Wolfe | Xue
Phage-assisted evolution and protein engineering yield compact, efficient prime editorsCellDoman et al.8/31/2137657419Liu
Polymer nanoparticles deliver mRNA to the lung for mucosal vaccinationScience Translational MedicineSuberi t al.8/16/2337585505Saltzman, Glazer
Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivoNature CommunicationsTao et al.8/15/2337582836Chen, Liu, Xu | Liu
Focused ultrasound-mediated brain genome editingProceedings of the National Academy of SciencesLao et al.8/14/2337579143Leong | Murray, Lutz
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathyThe Journal of Clinical InvestigationKabra et al.8/10/2337561581Gong, Emborg, Levine, Roy, Saha | Liu
Rational nanoparticle design: Optimization using insights from experiments and mathematical modelsJournal of Controlled ReleaseRichfield et al.7/22/2337442201Saltzman, Glazer
Therapeutic in vivo delivery of gene editing agentsCellRaguram et al.7/21/2335798006Liu
Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiencyNucleic Acids ResearchPonnienselvan et al.7/21/2337246708Sontheimer, Khvorova, Watts, Wolfe
Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrierPLOS BiologyHuang et al.7/19/2337467291Deverman | Heaney, Dickinson, Lagor
Potent and uniform fetal hemoglobin induction via base editingNature GeneticsMayuranathan et al.7/3/2337400614Liu | Tsai
Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cellsMolecular Therapy Nucleic AcidsJoynt et al.6/29/2337547293Liu
Adenine transversion editors enable precise, efficient A•T-to-C•G base editing in mammalian cells and embryosNature BiotechnologyChen et al.6/15/2337322276Liu
Template-jumping prime editing enables large insertion and exon rewriting in vivoNature CommunicationsDu et al.6/8/2337291100Gao, Anderson, Xue | Sontheimer
Liver directed adeno-associated viral vectors to treat metabolic diseaseJournal of inherited Metabolic DiseaseChuecos & Lagor6/5/2337254440Heaney, Dickinson, Lagor
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8Molecular TherapyDu et al.5/22/2337244253Chen, Liu, Xu
Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytesNature Biomedical EngineeringFoss et al.5/7/2337147433Wilson, Doudna
A Multifunctional and Highly Adaptable Reporter System for CRISPR/Cas EditingInternational Journal of Molecular SciencesWettengel et al.5/4/2337175977Hennebold
Efficient prime editing in mouse brain, liver and heart with dual AAVsNature BiotechnologyDavis et al.5/4/2337142705Liu
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in miceNature Biomedical EngineeringLiu et al.4/17/2337069266Liu
Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult miceProceedings of the National Academy of SciencesChen et al.4/17/2337068229Chen, Liu, Xu
Base editing rescue of spinal muscular atrophy in cells and in miceScienceArbab et al.3/30/202336996170Liu | Gao
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editingNature BiotechnologyLi et al.3/30/202336997680Gao, Anderson, Xue
Strategies for Improving Vascularization in Kidney Organoids: A Review of Current TrendsBiologyKonoe and Morizane3/26/2337106704Morizane, Lewis, Sabbisetti
CaMKIIδ gene editing- A base hit for the heartJournal of Cardiovascular AgingWalkey and Lagor3/22/202337065676Heaney, Dickinson, Lagor
Mitochondrial Base Editing: Recent Advances towards Therapeutic OpportunitiesInternational Journal of Molecular SciencesKar et al.3/18/202336982871Ekker, Clark
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in miceNature MedicineReichart et al.2/16/202336797483Liu
Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR DeliveryNano LettersYoshinaga et al.2/8/202336648291Leong
The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in VivoNano LettersRadmand et al.2/8/202336701517Dahlman, Santangelo
Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signalingAmerican Journal of Physiology - Cell PhysiologyCarrisoza-Gaytan et al.2/6/202336745528Morizane, Lewis, Sabbisetti
In vivo editing of the pan-endothelium by immunity evading simian adenoviral vectorBiomedicine & PharmacotherapyLorincz et al.2/1/202336587560Heaney, Dickinson, Lagor | Curiel
Focused ultrasound-mediated brain genome editingResearch SquareLao et al.1/20/202336712096Leong | Murray, Lutz
Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene TherapiesACS Synthetic BiologyCollins et al.1/20/202336627108Curiel
Reframing the perception of outliers and negative data in translational researchBrain Research BulletinMarina E. Emborg1/19/202336464129Gong, Emborg, Levine, Roy, Saha
Clinical progress in genome-editing technology and in vivo delivery techniquesTrends in GeneticsJennifer Khirallah et al.1/18/202336669950Chen, Liu, Xu
Accounting for diversity in the design of CRISPR-based therapeutic genome editingNature GeneticsKrishanu Saha1/2/202336593304Saha, Gamm, Roy, Skala
Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteinsBiomaterialsMetzger et al.12/13/202236527789Gong, Emborg, Levine, Roy, Saha | Murray, Lutz
In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled CellsAdvanced Functional MaterialsBulte et al.12/9/202236589903Bulte
Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA deliveryJournal of Controlled ReleaseDa Silva Sanchez et al.11/30/202236423872Dahlman, Santangelo
Diverse virus-encoded CRISPR-Cas systems include streamlined genome editorsCellAl-Sheyab et al.11/23/202236423580Doudna, Banfield
Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivoNature CommunicationsWillis et al.11/23/202236418298Liu
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activityNature BiotechnologyNeugebauer et al.11/10/202236357719Liu
Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney diseaseNature GeneticsXu et al.10/27/202236303074Freedman
Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecovirusesScienceMao et al.10/27/202236302057Saltzman, Glazer
Nucleic Acid Delivery to the Vascular EndotheliumMoleculare PharmaceuticsReschke et al.10/17/202236251765Saltzman, Glazer
Noninvasively differentiating acute and chronic nephropathies via multiparametric urea-CEST, nuclear Overhauser enhancement-CEST, and quantitative magnetization transfer MRIMagnetic Resonance in MedicineShin et al.10/13/202236226662Vandsburger
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept studyMolecular Therapy Methods & Clinical DevelopmentDoerfler et al.10/13/202236381302Bao, Lagor
Translating musculoskeletal bioengineering into tissue regeneration therapiesScience Translational MedicineKhodabukus et al.10/12/202236223445Gerbach, Bursac, Truskey
EZ Clear for simple, rapid, and robust mouse whole organ clearingeLifeHsu et al.10/11/202236218247Heaney, Dickinson, Lagor
Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applicationsNature CommunicationsAlbert et al. 10/11/202236220817Glazer, Ly, Saltzman
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editingNature CommunicationsGonzalez et al. 10/10/202236210364Asokan
Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discoveryScience AdvancesHiratsuka et al. 9/23/202236129975Morizane, Lewis, Sabbisetti
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMsNature BiotechnologyHuang et al.9/8/202236076084Liu
Efficient Homology-Directed Repair with Circular Single-Stranded DNA DonorsThe CRISPR JournalIyer et al.9/7/202236070530Sontheimer, Khvorova, Watts, Wolfe
Promoting validation and cross-phylogenetic integration in model organism researchDisease Models & MechanismsCheng et al.9/1/202236125045Heaney, Dickinson, Lagor | Ekker, Clark | Murray, Lutz
An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactorsJournal of Biological ChemistryEconomos et al.8/18/202235988651Glazer, Ly, Saltzman
Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivoNature CommunicationsNi et al.8/15/202235970837Dahlman, Santangelo
Designing and executing prime editing experiments in mammalian cellsNature ProtocolsDoman et al.8/8/202235941224Liu
Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genesThe American Association for the Study of Liver DiseasesRodríguez et al.8/6/202235932276Gao, Anderson, Xue | Sontheimer, Khvorova, Watts, Wolfe
Development of 68 Ga-Labeled Hepatitis E Virus Nanoparticles for Targeted Drug Delivery and Diagnostics with PETMolecular PharmaceuticsLambidis et al.8/1/202235857429Lam, Cheng
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editorsNature Biomedical EngineeringDavis et al.7/28/202235902773Liu | Chaikof
Therapeutic in vivo delivery of gene editing agentsCellRaguram et al.7/21/202235798006Liu
Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA deliveryNature NanotechnologyDobrowolski et al.6/30/202235768613Dahlman, Santangelo
Efficient Genome Editing Achieved via Plug-and-Play Adenovirus Piggyback Transport of Cas9/gRNA Complex on Viral Capsid SurfaceACS NanoLu et al.6/24/202235749339Curiel
Technologies to Assess Drug Response and Heterogeneity in Patient-Derived Cancer OrganoidsAnnual Review of Biomedical EngineeringSkala et al.6/6/202235259932Saha, Gamm, Roy, Skala
Inhibition of B-cell lymphoma 2 family proteins alters optical redox ratio, mitochondrial polarization, and cell energetics independent of cell stateJournal of Biomedical OpticsGillette et al.5/28/202235643815Saha, Gamm, Roy, Skala
Imaging the fetal nonhuman primate brain with SV2A positron emission tomography (PET)European Journal of Nuclear Medicine and Molecular ImagingRossano et al.5/28/202235633376Tarantal, Hartigan-O'Connor, Segal
CRISPR Library Screening in Cultured CardiomyocytesMethods in Molecular BiologyDeLuca and Bursac5/27/202235618895Gerbach, Bursac, Truskey
Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in miceScience Translational MedicineHunt et al.5/25/202235412328Freedman
In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoidsBiomedical Optics ExpressSamimi et al.5/18/202235781966Saha, Gamm, Roy, Skala
Structural biology of CRISPR-Cas immunity and genome editing enzymesNature Reviews MicrobiologyWang et al.5/13/202235562427Doudna, Banfield
The clinical progress of mRNA vaccines and immunotherapiesNature BiotechnologyBarbier et al5/9/202235534554Gao, Anderson, Xue
Translational Utility of the Nonhuman Primate ModelBiological Psychiatry: Cognitive Neuroscience and NeuroimagingTarantal et al.5/6/202235283343Tarantal, Hartigan-O'Connor, Segal
Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cellsNature GeneticsMouri et al.5/5/202235513721Liu
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor CellsMethods in Molecular BiologyPark et al.5/5/202235507169Gao, Anderson, Xue
Physiology assays in human kidney organoidsAmerican Journal of Physiology - Renal PhysiologyFreedman5/4/202235379001Freedman
CRISPR-Cas9 bends and twists DNA to read its sequenceNature Structural & Molecular Biology Cofsky et al.4/29/202235422516Doudna, Banfield
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNAAdvanced MaterialsXie et al.4/28/202235119139Gong, Emborg, Levine, Roy, Saha
Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated ciliaNature Biomedical EngineeringCruz et al.4/27/202235478224Freedman
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome EditingACS PublicationsChen et al.4/21/202235446558Sontheimer, Khvorova, Watts, Wolfe
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome HeterogeneityHuman Gene TherapyTran et al.4/19/202235293222Gao, Anderson, Xue
Crystal structure of an RNA/DNA strand exchange junctionPLOS OneCofsky et al.4/18/202235436289Doudna, Banfield
Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cellsDevelopmentBredemeyer et al.4/15/202235178561Hinson
An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell linesSTAR ProtocolsKar et al.4/15/202235496789Ekker, Clark
A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous SystemNature Cardiovascular ResearchKrolak et al.4/13/202235571675Deverman
Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cellsBiomaterialsWang et al.4/7/202235633376Gersbach, Bursac, Truskey
Durability of transgene expression after rAAV gene therapyMolecular TherapyMuhuri et al.4/6/202235283274Gao, Anderson, Xue
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cellsPLOS OneLuks et al.4/6/202235385514Saltzman, Glazer
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degenerationNature CommunicationsChoi et al.4/5/202235383196Liu
CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNANature BiotechnologyMok et al.4/4/202235379961Liu
A split prime editor with untethered reverse transcriptase and circular RNA templateNature BiotechnologyLiu et al.4/4/202235379962Gao, Anderson, Xue | Sontheimer, Khvorova, Watts, Wolfe
Label-Free Imaging to Track Reprogramming of Human Somatic CellsGEN BiotechnologyMolugu et al.4/1/202235586336Saha, Gamm, Roy, Skala
Robust, Durable Gene Activation In Vivo via mRNA-Encoded ActivatorsACS NanoBeyersdorf et al.3/31/202235357116Gersbach | Dahlman, Santangelo
Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integraseScientific ReportsLow et al.3/31/202235361849Murray, Lutz
AAV-delivered suppressor tRNA overcomes a nonsense mutation in miceNatureWang et al.3/23/202235322228Gao, Anderson, Xue
Species-dependent in vivo mRNA delivery and cellular responses to nanoparticlesNature NanotechnologyHatit et al.3/17/202235132167Dahlman, Santangelo
rAAV-delivered PTEN therapeutics for prostate cancerMolecular Therapy - Nucleic AcidsAi et al.3/8/202234976432Gao, Anderson, Xue
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopyChemical CommunicationsEspinoza et al.3/3/202235170593Bankiewicz, Murthy
Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulationNucleic Acids Research Economos et al.3/3/202235235944Glazer, Ly, Saltzman
Clinical magnetic hyperthermia requires integrated magnetic particle imagingWiley Interdisciplinary ReviewsHealy et al.3/3/202235238181Bulte
In Vivo Gene Editing in Lipid and Atherosclerosis ResearchMethods in Molecular BiologyGiorgi et al.3/2/202235237996Heaney, Dickinson, Lagor
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challengesScience Translational MedicineSchmidt et al.3/2/202235235338Gong, Emborg, Levine, Roy, Saha
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repairScience Translational MedicineGupta et al.3/2/202235235339Morizane, Lewis, Sabbisetti
Design of therapeutic biomaterials to control inflammationNature Reviews MaterialsTu et al.2/28/202235251702Leong
Treating Cardiovascular Disease with Liver Genome EngineeringCurrent Atherosclerosis ReportsHurley and Lagor2/24/202235230602Heaney, Dickinson, Lagor
Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosisProceedings of the National Academy of SciencesQiu et al.2/22/202235173043Chen, Liu, Xu
Nonhuman Primates in Translational ResearchAnnual Review of Animal BiosciencesTarantal et al.2/15/202235167321Tarantal, Hartigan-O'Connor, Segal | Tarantal, Segal
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis DeficienciesTrends in Molecular MedicineYang et al.2/12/202233714697Gao, Anderson, Xue
A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell TrackingMolecular Imaging and BiologyShalaby et al.2/10/202235146614Ronald
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmosetNature NeuroscienceGoertsen et al.1/25/202234887588Feng
Detecting Cardiovascular Protein-Protein Interactions by Proximity ProteomicsCirculation ResearchKushner et al.1/21/202235050691Hinson
Genome-wide detection of CRISPR editing in vivo using GUIDE-tagNature CommunicationsLiang et al.1/21/202235064134Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced specificity tagmentation-assisted PCRMolecular Therapy Methods & Clinical DevelopmentRyu et al.1/19/202235211637Hennebold
Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and FunctionsCirculationRomano et al.1/18/202234905694Hinson
BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2Nature Cell BiologySamelson et al.1/13/202235027731Conklin
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteinsCellBanskota et al.1/11/202235021064Liu | Chaikof
Gene editing in a Myo6 semi-dominant mouse model rescues auditory functionMolecular TherapyXue et al. 1/5/202234174443Chen, Liu, Xu
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liverMolecular TherapyZheng et al.1/5/202234998953Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editingMolecular TherapyKnipping et al.1/5/202234737067Liu
Acid-Sensitive Surfactants Enhance the Delivery of Nucleic AcidsMolecular PharmacologyRøise et al.12/21/202134931518Bankiewicz, Murthy
CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cellsAdvanced Drug Delivery ReviewsBloomer et al.12/20/202134942274Chen, Liu, Xu
Magnetic Iron Oxide Nanoparticles for Biomedical ApplicationsCurrent Opinion in Biomedical EngineeringJiang et al.12/20/202135211662Bao, Lagor
The Coiled-Coil Forming Peptide (KVSALKE)5 Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of ProteinsAdvanced Healthcare MaterialsLi et al.12/16/202134861744Bankiewicz, Murthy
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editingNature BiotechnologyAnzalone et al.12/9/202134887556Liu
Light: A Magical Tool for Controlled Drug DeliveryAdvanced Functional MaterialsTao et al.12/1/202134483808Leong
The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In VivoThe CRISPR JournalSabharwal et al.11/30/202134847747Ekker, Clark
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic diseaseNature CommunicationsTsagkaraki et al.11/26/202134836963Sontheimer, Khvorova, Watts, Wolfe
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine modelsNature CommunicationsHakim et al.11/24/202134819506Gersbach, Bursac, Truskey | Gersbach | Asokan
Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populationsJCI InsightHelms et al.11/12/202134767537Freedman
Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodiesNature CommunicationsFenix et al.11/3/202134732726Conklin
In vivo somatic cell base editing and prime editingMolecular TherapyNewby and Liu11/3/202134509669Liu
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophyMolecular TherapyPickar-Oliver et al.11/3/202134509668Gersbach, Bursac, Truskey | Asokan
Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome EditingACS PublicationsQiu et al.11/2/202134668716Chen, Liu, Xu
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivoNature CommunicationsIbraheim et al.11/1/202134725353Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Enhanced prime editing systems by manipulating cellular determinants of editing outcomesCellChen et al.10/28/202134653350Liu
Neuromuscular Development and Disease: Learning From in vitro and in vivo ModelsFrontiers in Cell and Developmental BiologyFralish et al.10/27/202134778273Gersbach, Bursac, Truskey | Asokan
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airwayMolecular TherapyLiang et al.10/23/202134695545Gao, Anderson, Xue | Heaney, Dickinson, Lagor
Electrophoretic mobility shift as a molecular beacon-based readout for miRNA detectionBiosensors and BioelectronicsOliveira Jr et al. 10/21/202134062334Ghiran
5' modifications improve potency and efficacy of DNA donors for precision genome editingeLifeGhanta et al.10/19/202134665130Sontheimer, Khvorova, Watts, Wolfe
SnRNA sequencing defines signaling by RBC-derived extracellular vesicles in the murine heartLife Science AllianceValkov et al.10/18/202134663679Ghiran
Deletion and replacement of long genomic sequences using prime editingNature BiotechnologyJiang et al.10/14/202134650270Gao, Anderson, Xue
Disulfide-compatible phage-assisted continuous evolution in the periplasmic spaceNature CommunicationsMorrison et al.10/13/202134645844Liu
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editorsNucleic Acids ResearchKrishnamurthy et al.10/11/202134520545McCray | Liu
Kidney development to kidney organoids and back againSeminars in Cell & Developmental BiologyGupta and Morizane10/6/202134627669Morizane, Lewis, Sabbisetti
Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungsNature Biomedical EngineeringLokugamage et al.10/6/202134616046Dahlman, Santangelo
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Nucleic Acid Therapeutics for Neurological DiseasesNeurotherapeuticsWatts et al.4/29/201931037650Sontheimer, Khvorova, Watts, Wolfe
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-SeqScienceWienert et al.4/19/201931000663McDevitt
CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling.Genome MedicineMou et al.4/16/201930987660Gao, Anderson, Xue
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging.Nature Chemical BiologyOh et al.4/1/201930936501McDevitt
Kidney Organoids in Translational Medicine: Disease Modeling and Regenerative MedicineDevelopmental DynamicsMiyoshi et al.3/26/201935633376Morizane, Lewis, Sabbisetti
Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression.ImmunityMartinez-Navio et al.3/19/201930850342Gao, Anderson, Xue
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing AntibodiesMolecular TherapyGardner et al.3/6/201930704961Gao, Anderson, Xue
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome EditingMolecular CellEdraki et al.2/21/201930581144Gao, Anderson, Xue
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems.Bioconjugate ChemistryWang et al.2/20/201930592619Gong, Emborg, Levine, Roy, Saha
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous SystemMethods in Molecular BiologyGessler et al.2/20/201930783972Gao, Anderson, Xue
Quantitative and Digital Droplet-Based AAV Genome TitrationMethods in Molecular BiologySanmiguel et al.2/20/201930783968Gao, Anderson, Xue
Simultaneous targeting of linked loci in mouse embryos using base editingScientific ReportsLee et al.2/7/201930733567Liu
Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome EditingAccounts of Chemical ResearchChang et al.12/26/201830586281Chen, Liu, Xu
Illuminating the genome-wide activity of genome editors for safe and effective therapeuticsGenome BiologyCheng et al.12/22/201830577870Tsai
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene TransferMolecular Therapy: Methods & Clinical DevelopmentWang et al.12/14/201830397628Gao, Anderson, Xue
MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7Molecular Therapy: Methods & Clinical DevelopmentLuo et al.12/14/201830406154Gao, Anderson, Xue
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid NanoparticlesAdvanced Healthcare MaterialsYamin et al.11/22/201830565897Chen, Liu, Xu
Targeting fidelity of adenine and cytosine base editors in mouse embryosNature CommunicationsLee et al.11/15/201830442934Liu
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene TherapyHuman Gene TherapyLiang et al.11/13/201829893153Gao, Anderson, Xue
Understanding and repurposing CRISPR-mediated alternative splicingGenome BiologySmith et al.11/6/201830400804Gao, Anderson, Xue
Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq.Nature ProtocolsLazzarotto et al.10/19/201830341435Tsai
Cas9-mediated Allelic Exchange Repairs Compound Heterozygous Recessive Mutations in MiceNature BiotechnologyWang et al.10/1/201830102296Gao, Anderson, Xue
The temporal landscape of recursive splicing during Pol II transcription elongation in human cellsPLOS GeneticsZhang et al.8/27/201830148885Gao, Anderson, Xue