SCGE bioRxiv Channel
Publication TitleJournalAuthorsPub DatePubMedProject PIs
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
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
Engineered pegRNAs improve prime editing efficiencyNature BiotechnologyNelson et al.10/4/202134608327Liu
In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic CellsMolecular Imaging and BiologyBulte et al.9/28/202134581954Bulte
Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene TherapiesACS PublicationsCollins and Curiel9/28/202134415739Curiel
External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editorsBiomaterials ScienceXie et al.9/14/202134286726Gong, Emborg, Levine, Roy, Saha
Receptor Switching in Newly Evolved Adeno-associated VirusesJournal of VirologyHavlik et al.9/9/202134232726Asokan
Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorderNature Communicationsde Jong et al.9/1/202134471112Leong
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3Proceeding of the National Academy of Sciences in the United States of AmericaQiu et al.9/1/202133649229Chen, Liu, Xu
DNA interference states of the hypercompact CRISPR-CasΦ effectorNature Structural & Molecular BiologyPausch et al.8/28/202134381246Doudna, Banfield
Easy-Prime: a machine learning-based prime editor design toolGenome BiologyLi et al.8/19/202134412673Tsai
Transgenic mice for in vivo epigenome editing with CRISPR-based systemsNature MethodsGemberling et al.8/18/202134341582Gersbach | Asokan
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2EFrontiers in Cell and Developmental BiologyFeliciano et al.8/16/202135633376Conklin
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticlesJournal of Controlled ReleaseWang et al.8/10/202134174352Gong, Emborg, Levine, Roy, Saha
Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2Cell ReportsLadha et al.8/10/202134380038Hinson
Vector Strategies to Actualize B Cell-Based Gene TherapiesJournal of ImmunologyJeske et al.8/1/202134321286Curiel
Label-free imaging for quality control of cardiomyocyte differentiationNature CommunicationsQian et al.7/28/202134321477Saha, Gamm, Roy, Skala
Recent innovations in fluorescence lifetime imaging microscopy for biology and medicineJournal of Biomedical OpticsDatta et al.7/26/202134247457Saha, Gamm, Roy, Skala
Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOSAging CellGete et al.7/20/202134086398Liu
Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical ScienceAnnual Review of Biomedical EngineeringAbdeen et al.7/13/202133909475Gersbach | Gersbach, Bursac, Truskey
Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehiclesJournal of Controlled ReleaseXu et al.7/10/202134077782Saltzman, Glazer
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progressionGene TherapyAi et al.7/6/202134226687Gao, Anderson, Xue
Targeting the De Novo Purine Synthesis Pathway Through Adenylosuccinate Lyase Depletion Impairs Liver Cancer Growth by Perturbing Mitochondrial FunctionHepatologyJiang et al.7/1/202133336367Gao, Anderson, Xue
Base editing of haematopoietic stem cells rescues sickle cell disease in miceNatureNewby et al.7/1/202134079130Liu | Tsai
Mucopolysaccharidoses type I gene therapyJournal of Inherited Metabolic DiseaseHurt et al.6/29/202134189746Curiel
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learningNature BiotechnologyKoblan et al.6/28/202134183861Liu | Chaikof
3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory considerationClinical and Translational ScienceWang et al.6/16/202133982436Freedman
Induced pluripotent stem cells provide mega insights into kidney diseaseKidney InternationalLuciani et al.6/15/202132571490Freedman
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapyMolecular Therapy Methods & Clinical DevelopmentMarquez Loza et al.6/11/202133768133McCray
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repairCellWilde et al.6/10/202134043941Feng
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation by activation of acid sphingomyelinaseBloodWang et al.6/1/202134075401Yi
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafnessScience Translational MedicineYeh et al.6/1/202132493795Liu | Chen, Liu, Xu
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineeringCell ReportsHamilton et al.6/1/202134077734Doudna, Banfield
Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem CellsGenes (Basel)Liu et al.5/25/202134070435Chen, Liu, Xu
Quantification of Cellular Densities and Antigenic Properties using Magnetic LevitationJournal of Visualized ExperiementsThompson et al.5/17/202134057455Ghiran
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Frontiers in ImmunologyZhan et al.5/11/202134046041Gao, Anderson, Xue
Three-dimensional tissue-engineered human skeletal muscle model of Pompe diseaseCommunications BiologyWang et al.5/5/202133953320Gersbach, Bursac, Truskey
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftmentCell ReportsPettinato et al.5/4/202133951429Hinson
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell CarcinomaJournal of the National Cancer InstituteShearer et al.5/4/202132944748Morizane, Lewis, Sabbisetti
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic NanovaccinesAdvanced Science (Weinh)Hu et al.5/1/202134386315Leong
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable TransductionFrontiers in ImmunologyMuhri et al.4/28/202133995418Gao, Anderson, Xue
Targeting the Apoa1 locus for liver-directed gene therapyMolecular Therapy Methods & Clinical DevelopmentDe Giorgi et al.4/24/202134141821Bao, Lagor | Heaney, Dickinson, Lagor
Improved prime editors enable pathogenic allele correction and cancer modelling in adult miceNature CommunicationsLiu et al.4/9/202133837189Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
The NIH Somatic Cell Genome Editing programNatureSaha, K., Sontheimer, E.J., Brooks, P.J. et al.4/7/202133828315SCGE Consortium
A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integrationNucleic Acids ResearchJakhanwal et al.4/6/202133693715Doudna, Banfield
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector ImpuritiesFrontiers in ImmunologyRumachik et al.4/1/202133868302McDevitt
Large-scale molecular epidemiological analysis of AAV in a cancer patient populationOncogeneQin et al.3/29/202133782545Gao, Anderson, Xue
PEGylation of poly(amine-co-ester) polyplexes for tunable gene deliveryBiomaterialsGrun et al3/24/202133813260Saltzman, Glazer
Loss of Sarcomeric Proteins via Upregulation of JAK/STAT Signaling Underlies Interferon-γ-induced Contractile Deficit in Engineered Human MyocardiumActa BiomaterialiaZhan et al.3/8/202135633376Gersbach, Bursac, Truskey
Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expressionGenome BiologyGao et al.3/16/202133722289Tsai | Liu
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19Science Translational MedicinePerez-Bermejo et al.3/15/202133723017McDevitt
Nanoparticles for delivery of agents to fetal lungsActa BiomaterialiaUllrich et al.3/15/202133484911Saltzman, Glazer
Therapeutic RNA Delivery for COVID and Other DiseasesAdvanced Healthcare MaterialsDobrowolski et al.3/4/202133661555Dahlman, Santangelo
Magnetic Forces Enable Control of Biological Processes In VivoJournal of Applied Mechanics Bao3/1/202134168385Bao, Lagor
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease.Frontiers in PhysiologyKhodabukus2/26/202133716768Gersbach, Bursac, Truskey
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interesteLifeChen et al.2/23/202133620031Deverman
Massively parallel assessment of human variants with base editor screensCellHanna et al.2/18/202133606977Liu
Precision genome editing using cytosine and adenine base editors in mammalian cellsNature ProtocolsHuang et al.2/16/202133462442Liu
PrimeDesign software for rapid and simplified design of prime editing guide RNAsNature CommunicationsHsu et al.2/15/202133589617Liu
Soluble CD40L activates soluble and cell-surface integrin αvβ3, α5β1, and α4β1 by binding to the allosteric ligand-binding site (site 2)Journal of Biological ChemistryTakada et al.2/9/202133571526Lam, Cheng
Viral vector platforms within the gene therapy landscape.Signal Transduction and Targeted TherapyBulcha et al.2/8/202133558455Gao, Anderson, Xue
Gene-based therapies for neurodegenerative diseasesNature NeuroscienceSun et al.2/1/202133526943Gong, Emborg, Levine, Roy, Saha
Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver OrganoidsCell SystemsVelazquez et al.1/20/2021332907415Kiani
Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routesPharmaceutical Patent AnalysisChen et al.1/20/202133467938Lam, Cheng
Engineering highly efficient backsplicing and translation of synthetic circRNAsMolecular Therapy - Nucleic Acids JournalMeganck et al.1/16/2021336142328Asokan
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