SCGE bioRxiv Channel
Publication TitleJournalAuthorsPub DatePubMedProject PIs
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor CellsMethods in Molecular BiologyPark et al5/5/202235507169Gao
CRISPR-Cas9 bends and twists DNA to read its sequenceNature Structural & Molecular Biology Cofsky et al4/29/202235422516Doudna
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome EditingACS PublicationsChen et al4/21/202235446558Sontheimer, Khvorova, Watts, Wolfe
Crystal structure of an RNA/DNA strand exchange junctionPLOS OneCofsky et al4/18/202235436289Doudna
An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell linesSTAR ProtocolsKar et al4/15/202235496789Ekker, Clark
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degenerationNature CommunicationsChoi et al4/5/202235383196Liu
CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNANature BiotechnologyMok et al4/4/202235379961Liu
A split prime editor with untethered reverse transcriptase and circular RNA templateNature BiotechnologyLiu et al4/4/202235379962Xue, Sontheimer
Robust, Durable Gene Activation In Vivo via mRNA-Encoded ActivatorsACS NanoBeyersdorf et al3/31/202235357116Gersbach | Dahlman, Santangelo
Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integraseScientific ReportsLow et al3/31/202235361849Murray, Lutz
AAV-delivered suppressor tRNA overcomes a nonsense mutation in miceNatureWang et al3/23/202235322228Gao, Anderson, Xue
Species-dependent in vivo mRNA delivery and cellular responses to nanoparticlesNature NanotechnologyHatit et al3/17/202235132167Dahlman, Santangelo
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopyChemCommEspinoza et al 3/3/202235170593Murthy
Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulationNucleic Acids Research Economos et al3/3/202235235944Glazer, Ly, Saltzman
Clinical magnetic hyperthermia requires integrated magnetic particle imagingWiley Interdisciplinary ReviewsHealy et al3/3/202235238181Bulte
In Vivo Gene Editing in Lipid and Atherosclerosis ResearchMethods in Molecular BiologyGiorgi et al3/2/202235237996Lagor
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challengesScience Translational MedicineSchmidt et al3/2/202235235338Gong, Emborg, Levine, Roy, Saha
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repairScience Translational MedicineGupta et al3/2/202235235339Morizane, Lewis, Sabbisetti
Design of therapeutic biomaterials to control inflammationNature ReviewTu et al2/28/202235251702Leong
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 al2/22/202235173043Chen, Liu, Xu
Nonhuman Primates in Translational ResearchAnnual Review of Animal BiosciencesTarantal et al2/15/202235167321Tarantal
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis DeficienciesTrends in Molecular MedicineYang et al2/12/202233714697Gao
A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell TrackingMolecular Imaging and BiologyShalaby et al2/10/202235146614Ronald
Gene editing in a Myo6 semi-dominant mouse model rescues auditory functionMolecular TherapyXue et al 1/5/202234174443Chen
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liverMolecular TherapyZheng et al1/5/202234998953Wolfe, Xue
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNAAdvanced MaterialsXie et al2/4/202235119139Gong
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmosetNature NeuroscienceGoertsen et al1/25/202234887588Feng
Detecting Cardiovascular Protein-Protein Interactions by Proximity ProteomicsCirculation ResearchKushner et al1/21/202235050691Hinson
Genome-wide detection of CRISPR editing in vivo using GUIDE-tagNature CommunicationsLiang et al1/21/202235064134Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteinsCellBanskota et al1/20/202235021064Liu | Chaikof
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced specificity tagmentation-assisted PCRMolecular Therapy Methods & Clinical DevelopmentRyu et al1/19/202235211637Hennebold
Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and FunctionsCirculationRomano et al1/18/202234905694Hinson
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 al1/5/202234737067Liu
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical ApplicationsMaterials Today (Kidlington England)Hu H et al1/1/202234421329Leong
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 al12/20/202135211662Bao
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editingNature BiotechnologyAnzalone et al12/9/202134887556Liu
Light: A Magical Tool for Controlled Drug DeliveryAdvanced Functional MaterialsTao Y et al12/1/202134483808Leong
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 al11/24/202134819506Gersbach, Bursac, Truskey | Gersbach | Asokan
Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populationsJCI InsightHelms et al11/12/202134767537Freedman
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 al11/3/202134509668Gersbach, Bursac, Truskey | Asokan
Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome EditingACS PublicationsQiu M 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 al10/27/202134778273Gersbach
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airwayMolecular TherapyLiang et al10/23/202134695545Gao, 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 T, 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 S, 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 al10/4/202134608327Liu
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
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 P, et al.8/28/202134381246Doudna, Banfield
Easy-Prime: a machine learning-based prime editor design toolGenome BiologyLi et al.8/19/202234412673Tsai
Transgenic mice for in vivo epigenome editing with CRISPR-based systemsNature MethodsGemberling et al.8/18/202134341582Gersbach | Asokan
A pH-Responsive Silica–Metal–Organic Framework Hybrid Nanoparticle for the Delivery of Hydrophilic Drugs, Nucleic Acids, and CRISPR-Cas9 Genome-Editing MachineriesJournal of Controlled ReleaseWang et al.8/10/202132380204Gong, Emborg, Levine, Roy, Saha
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 al7/20/202134086398Liu
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 GA, et al.7/1/202134079130Liu | Tsai
Mucopolysaccharidoses type I gene therapyJournal of Inherited Metabolic DiseaseHurt, SC6/29/202134189746Curiel
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learningNature BiotechnologyKoblan LW, et al.6/28/202134183861Liu | Chaikof
Induced pluripotent stem cells provide mega insights into kidney diseaseKidney InternationalLuciani A, 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 LI, et al.6/11/202133768133McCray
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repairCellWilde JJ, et al.6/10/202134043941Feng
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation by activation of acid sphingomyelinaseBloodWang J, et al.6/1/202134075401Yi
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineeringCell ReportsHamilton JR, et al.6/1/202134077734Doudna, Banfield
Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem CellsGenes (Basel)Liu X, et al5/25/202134070435Chen, Liu, Xu
Quantification of Cellular Densities and Antigenic Properties using Magnetic LevitationJournal of Visualized ExperiementsThompson L, et al.5/17/202134057455Ghiran
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Frontiers in ImmunologyWei Zhan, et al.5/11/202134046041Gao, Anderson, Xue
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftmentCell ReportsAnthony M Pettinato, et al.5/4/202133951429Hinson
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell CarcinomaJournal of the National Cancer InstituteShearer JJ, et al.5/4/202132944748Morizane, Lewis, Sabbisetti
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic NanovaccinesAdvanced Science (Weinh)Hu H, 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 al4/28/202133995418Gao
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 P, 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 S, et al.4/6/202133693715Doudna, Banfield
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector ImpuritiesFrontiers in ImmunologyRumachik, Neil G, et al.4/1/202133868302McDevitt
Large-scale molecular epidemiological analysis of AAV in a cancer patient populationOncogeneQin W, et al.3/29/202133782545Gao, Anderson, Xue
PEGylation of poly(amine-co-ester) polyplexes for tunable gene deliveryBiomaterialsGrun et al3/24/202133813260Saltzman
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19Science Translational MedicinePerez-Bermejo JA, et al.3/15/202133723017McDevitt
Nanoparticles for delivery of agents to fetal lungsActa BiomaterialiaUllrich SJ, et al.3/15/202133484911Saltzman, Glazer
Therapeutic RNA Delivery for COVID and Other DiseasesAdvanced Healthcare MaterialsDobrowolski C, et al.3/4/202133661555Dahlman, Santangelo
Magnetic Forces Enable Control of Biological Processes In VivoJournal of Applied Mechanics Bao3/1/202134168385Bao
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease.Frontiers in PhysiologyKhodabukus A.2/26/202133716768Gersbach, Bursac, Truskey
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interestElifeChen AT, et al.2/23/202133620031Deverman
Massively parallel assessment of human variants with base editor screensCellHanna et al2/18/202133606977Liu
Precision genome editing using cytosine and adenine base editors in mammalian cellsNature ProtocolsHuang, TP, et al.2/16/202133462442Liu
PrimeDesign software for rapid and simplified design of prime editing guide RNAsNature CommunicationsHsu, Jonathan Y, 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 YK, et al.2/9/202133571526Lam, Cheng
Viral vector platforms within the gene therapy landscape.Signal Transduction and Targeted TherapyBulcha, Jote T, et al.2/8/202133558455Gao, Anderson, Xue
Gene-based therapies for neurodegenerative diseasesNature NeuroscienceSun, Jichao, 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 JJ, et al.1/20/2021332907415Kiani
Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routesPharmaceutical Patent AnalysisChen, CC, et al.1/20/202133467938Lam, Cheng
Engineering highly efficient backsplicing and translation of synthetic circRNAsMolecular Therapy - Nucleic Acids JournalMeganck, RM, et al.1/16/2021336142328Asokan
Tools for experimental and computational analyses of off-target editing by programmable nucleasesNature ProtocolsBao, X Robert, et al.1/16/202133288953Bao
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscleScience AdvancesChen Z, et al.1/7/202133523949Gersbach, Bursac, Truskey
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.NatureKloban, AL, et al.1/6/202133408413Liu
Overcoming innate immune barriers that impede AAV gene therapy vectorsThe Journal of Clinical InvestigationMuhuri, M, et al.1/4/202133393506Gao, Anderson, Xue
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue ConstructsMethods in Molecular BiologyHeifer, Abbigail, et al.1/1/202132857373Gersbach, Bursac, Truskey
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical ApplicationsMater Today (Kidlington)Hu H, et al.1/1/202134421329Leong
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial functionHepatologyJiang, Tingting, et al.12/17/202033336367Gao, Anderson, Xue
Multicellular Systems to Translate Somatic Cell Genome Editors to HumansCurrent Opinion in BiotechnologyHernandez-Gordillo V, et al.12/16/202033718690Kiana
The dawn of non-human primate models for neurodevelopmental disorders.Current Opinion in Genetics and DevelopmentAida, Tomoni, et al..12/1/202033718690Feng
Advances in microphysiological blood-brain barrier (BBB) models towards drug deliveryCurrent Opinion in BiotechnologyLee, Caleb S, et al.12/1/202032711361Leong
Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation ScreensCell ReportsBlack, Joshua B, et al.12/1/202033264623Gersbach
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis.Molecular CellLeclair, Nathan K, et al.11/19/202033176162Hinson
Phage-assisted continuous and non-continuous evolutionNature ProtocolsMiller, Shannon M, et al.11/16/202033199872Liu
The once and future gene therapyNature CommunicationsBulaklak, Karen, et al.11/16/202033199717Gersbach, Bursac, Truskey | Gersbach
Delayed urea differential enhancement CEST (dudeCEST)-MRI with T 1 correction for monitoring renal urea handlingMagnetic Resonance in MedicineShin, Soo Hyun, et al.11/12/202033180343Vandsburger
Advances in Alpha-1 Antitrypsin Gene TherapyAmerica Journal of Respiratory Cell and Nolecular BiologyLorincz, Reka, et al.11/1/202032668173Curiel
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.Journal of VirologyMadigan, Victoria J, et al.10/14/202032817219Asokan
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the LiverAdvanced MaterialsGong, Jing, et al.10/14/202033053221Leong
3D kidney organoids for bench-to-bedside translationJournal of Molecular MedicineGupta, Navin, et al.10/9/202033034708Morizane, Lewis, Sabbisetti
Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV VectorsbioRxivIbraheim, Raed, et al.10/9/2020PreprintSontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 VariantsCirculationPettinato, Anthony M, et al.10/7/202033025817Hinson
Opportunities and limitations of genetically modified nonhuman primate models for neuroscience researchProceeding of the National Academy of Sciences in the United States of AmericaFeng, Guoping, et al.9/29/202032817435Feng
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular DystrophyMolecular Therapy Methods & Clinical DevelopmentKwon, Jennifer B, et al.9/28/202033145368Asokan | Gersbach, Bursac, Truskey
COVID-19 CG:Tracking SARS-CoV-2 mutations by locations and dates of interestbioRxivChen, Albert Tian, et al.9/28/202032995794Deverman
Synthetic immunomodulation with a CRISPR super-repressor in vivoNature Cell BiologyMoghadam, Farzaneh, et al.9/22/202032884147Kiani
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided ApproachJournal of VirologyHavalik, Patrick L, et al.9/15/202032669336Asokan
SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patientsbioRxivPérez-Bermejo, Juan A, et al.9/12/202032884147McDevitt
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In VivoMolecular Therapy Methods & Clinical DevelopmentZhang, Kun, et al.9/11/202032953927Chen, Liu, Xu
Rapid, precise quantification of large DNA excisions and inversions by ddPCR.Scientific ReportsWatry, Hannah L, et al.9/10/202032913194McDevitt
New Directions in Pulmonary Gene TherapyHuman Gene TherapyVu, A et al.9/3/202032814451McCray
A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries.Journal of Controlled ReleaseWang, Yuyuan, et al.8/10/202032380204Gong, Emborg, Levine, Roy, Saha
A scoutRNA Is Required for Some Type V CRISPR-Cas SystemsMolecular CellHarrington, Lucas B, et al.8/6/202032645367Doudna, Banfield
NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer.CellsHuang, Tung-Yung, et al.8/3/202032756527Lam, Cheng
Finding order in slow axonal transportCurrent Opinion in NeurobiologyWang, Yuyuan, et al.8/1/202032361600Gong, Emborg, Levine, Roy, Saha
DNA capture by a CRISPR-Cas9-guided adenine base editor.ScienceAl-Shayeb, Basem, et al.7/31/202032732424Doudna, Banfield | Liu
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury.Science AdvancesEttou, Sandrine, et al.7/24/202032754639Morizane, Lewis, Sabbisetti
Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning.CellArbab, Mandana, et al.7/23/202032533916Liu
CRISPR-CasΦ from huge phages is a hypercompact genome editor.SciencePausch, Patrick, et al.7/17/202032675376Doudna, Banfield
Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery.Bioconjugate ChemistryLi, Yamin, et al.7/15/202032520527Chen, Liu, Xu
Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers.Toxicological SciencesKhodabukus, Alastair, et al.7/1/202032294208Gersbach, Bursac, Truskey
The delivery challenge: fulfilling the promise of therapeutic genome editingNature Biotechnologyvan Haasteren, Joost, et al.6/29/202032601435Bankiewicz, Murthy
Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase.Nature BiotechnologyWilson, Christopher, et al.6/29/202032601430Liu
Polymeric vehicles for nucleic acid delivery.Advance Drug Delivery ReviewsPiotrowski-Daspit, Alexandra S, et al.6/23/202032585159Saltzman, Glazer
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors.Nature BiotechnologyAnzalone, Andrew V, et al.6/22/202032572269Liu
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing.NatureMok, Beverly Y, et al.6/22/202032641830Liu
CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity.Nature BiotechnologyLazzarotto, Cicera R, et al.6/15/202032541958Tsai
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells.Cell Stem CellNagpal, Neha, et al.6/4/202032320679Sontheimer, Khvorova, Watts, Wolfe
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.Molecular TherapyLi, Ang, et al.6/3/202032348718Bao, Lagor | Heaney, Dickinson, Lagor
Can we mimic skeletal muscles for novel drug discovery?Expert Opinion on Drug DeliveryBroer, Torie, et al.6/1/202032122176Gersbach, Bursac, Truskey
Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligandsBioengineering & Translational MedicineGan Z, et al.5/27/202033758781Dahlman, Santangelo
YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells.HepatologySmith, Jordan L, et al.5/26/202032452550Gao, Anderson, Xue | Liu
The developing toolkit of continuous directed evolutionNature Chemical BiologyMorrison, Mary S, et al.5/22/202032444838Liu
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct VectorsMolecular Therapy, Methods & Clinical DevelopmentRumachik, Neil G, et al.5/22/202032995354McDevitt
Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles.Angewandte Chemie International Edition in EnglishLi, Yamin, et al.5/21/202032438474Chen, Liu, Xu
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional ActivatorsStem Cell ReportsKwon, Jennifer B, et al.5/12/202032330446Gersbach, Bursac, Truskey | Asokan | Gersbach
Advances in genome editing for genetic hearing loss.Advance Drug Delivery ReviewsDing, Ning, et al.5/7/202032387678Bao, Lagor
Once upon a dish: engineering multicellular systemsDevelopmentHaase, Kristina, et al.5/4/202032366376Freedman
Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulationNature CommunicationsSeo, Jai Woong, et al.4/30/202032355221Lam, Cheng
Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope.Nature CommunicationsJiang, Tingting, et al.4/24/202032332735Gao, Anderson, Xue | Liu
Prime genome editing in rice and wheatNature BiotechnologyLin, Qiupeng, et al.3/16/202032393904Liu
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity.Nature BiotechnologyRichter, Michelle F, et al.3/16/202032433547Liu
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic DiseasesTheranosticsWu, Shao-Shuai, et al.3/15/202032292501Gao, Anderson, Xue
Targeted Delivery of Secretory Promelittin via Novel Poly(lactone‐co‐β‐amino ester) Nanoparticles for Treatment of Breast Cancer Brain MetastasesAdvanced ScienceZhou, Yu, et al.3/7/202032154067Zhou
Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics.Kidney360Liu, Esther, et al.3/1/202032656538Freedman
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applicationsBiomaterialsLi, Yamin, et al.3/1/202031945616Chen, Liu, Xu
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