SCGE bioRxiv Channel
Publication TitleAuthorsJournalPub DatePubMedProject PIs
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications Hu H, et al.Materials Today (Kidlington England)1/1/2234483808Leong
Light: A Magical Tool for Controlled Drug DeliveryTao Y, et al.Advanced Functional Materials12/1/2134483808Leong
External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors Xie R, et al.Biomaterials science9/14/2134286726Gong, Emborg, Levine, Roy, Saha
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editorsKrishnamurthy S, et al.Nucleic Acids Research9/14/2134520545McCray | Liu
Receptor Switching in Newly Evolved Adeno-associated Viruses Havlik LP, et al.Journal of Virology9/9/2134232726Asokan
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3Qiu M, et al.Proceeding of the National Academy of Sciences in the United States of America9/1/2133649229Chen, Liu, Xu
DNA interference states of the hypercompact CRISPR-CasΦ effector Pausch P, et al.Nature Structural & Molecular Biology8/28/2133649229Doudna, Banfield
Transgenic mice for in vivo epigenome editing with CRISPR-based systemsGemberling MP, et al.Nature Methods8/18/2134341582Gersbach, Bursac, Truskey
A pH-Responsive Silica–Metal–Organic Framework Hybrid Nanoparticle for the Delivery of Hydrophilic Drugs, Nucleic Acids, and CRISPR-Cas9 Genome-Editing MachineriesWang Y, et al.J Control Release8/10/2132380204Gong, Emborg, Levine, Roy, Saha
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles.Wang Y, et al.J Control Release8/10/2134174352Gong, Emborg, Levine, Roy, Saha
Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2 Ladha FA, et al.Cell Reports8/10/2134380038Hinson
Vector Strategies to Actualize B Cell-Based Gene Therapies Jeske AM, et al.Journal of Immunology8/1/2134321286Curiel
Label-free imaging for quality control of cardiomyocyte differentiationQian T, et al.Nature Communications7/28/1134321477Saha, Gamm, Roy, Skala
Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine Datta R, et al.Journal of Biomedical Optics7/26/2134247457Saha, Gamm, Roy, Skala
Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science Abdeen AA, et al.Annu Rev Biomed Eng. 7/13/2133909475Gersbach
Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehiclesXu E, et al.J Control Release7/10/2134077782Saltzman, Glazer
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progressionAi J, et al.Gene Therapy7/6/2134226687Gao, Anderson, Xue
Targeting the De Novo Purine Synthesis Pathway Through Adenylosuccinate Lyase Depletion Impairs Liver Cancer Growth by Perturbing Mitochondrial FunctionJiang T, et al.Hepatology7/1/2133336367Gao, Anderson, Xue
Base editing of haematopoietic stem cells rescues sickle cell disease in miceNewby GA, et al.Nature7/1/2134079130Liu
Mucopolysaccharidoses type I gene therapyHurt, SCJournal of inherited metabolic disease6/29/2134189746Curiel
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learningKoblan LW, et al.Nature Biotechnology6/28/2134183861Liu, Chaikof
Induced pluripotent stem cells provide mega insights into kidney diseaseLuciani A, et al.Kidney International6/15/2132571490Freedman
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy Marquez Loza LI, et al.Molecular Therapy Methods & Clinical Development6/11/2133768133McCray
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair Wilde JJ, et al.Cell6/10/2134043941Feng
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation by activation of acid sphingomyelinase Wang J, et al.Blood6/1/2134075401Yi
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering Hamilton JR, et al. Cell Reports6/1/2134077734Doudna, Banfield
Quantification of Cellular Densities and Antigenic Properties using Magnetic LevitationThompson L, et al.Journal of Visualized experiements5/17/2134057455Ghiran
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Wei Zhan, et al.Frontiers in Immunology5/11/2134046041Gao, Anderson, Xue
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftmentAnthony M Pettinato, et al.Cell Reports5/4/2133951429Hinson
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma Shearer JJ, et al.National Cancer Inst.5/4/2132944748Morizane, Lewis, Sabbisetti
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines Hu H, et al.Advanced Science (Weinh)5/1/2134386315Leong
Targeting the Apoa1 locus for liver-directed gene therapyDe Giorgi, et al.Molecular Therapy Methods & Clinical Development 4/24/2134141821Bao, Lagor
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice Liu P, et al.Nature Communications4/9/2133837189Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
The NIH Somatic Cell Genome Editing programSaha, K., Sontheimer, E.J., Brooks, P.J. et al.Nature4/7/2133828315SCGE Consortium
A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration Jakhanwal S, et al.Nucleic Acids Research4/6/2133693715Doudna, Banfield
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector ImpuritiesRumachik, Neil G, et al.Frontiers in Immunology4/1/2133868302McDevitt
Large-scale molecular epidemiological analysis of AAV in a cancer patient population Qin W, et al.Oncogene3/29/2133782545Gao, Anderson, Xue
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19 Perez-Bermejo JA, et al.Science Translational Medicine3/15/2133723017McDevitt
Nanoparticles for delivery of agents to fetal lungsUllrich SJ, et al.Acta Biomaterialia3/15/2133484911Saltzman, Glazer
Therapeutic RNA Delivery for COVID and Other Diseases Dobrowolski C, et al.Advanced Healthcare Materials3/4/2133661555Dahlman, Santangelo
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease.Khodabukus A.Frontiers in Physiology2/26/2133716768Gersbach, Bursac, Truskey
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interestChen AT, et al.Elife2/23/2133620031Deverman
Precision genome editing using cytosine and adenine base editors in mammalian cells Huang, TP, et al.Nature Protocols2/16/2133462442Liu
PrimeDesign software for rapid and simplified design of prime editing guide RNAs Hsu, Jonathan Y, et al.Nature Communications2/15/2133589617Liu
Soluble CD40L activates soluble and cell-surface integrin αvβ3, α5β1, and α4β1 by binding to the allosteric ligand-binding site (site 2)Takada YK, et al.Journal of Biological Chemistry2/9/2133571526Lam, Cheng
Viral vector platforms within the gene therapy landscape.Bulcha, Jote T, et al.Signal Transduction and Targeted Therapy2/8/2133558455Gao, Anderson, Xue
Gene-based therapies for neurodegenerative diseasesSun, Jichao, et al.Nature Neuroscience2/1/2133526943Gong, Emborg, Levine, Roy, Saha
Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver Organoids Velazquez JJ, et al.Cell Systems1/20/21332907415Kiani
Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routesChen, CC, et al.Pharmaceutical Patent Analysis1/20/2133467938Lam, Cheng
Engineering highly efficient backsplicing and translation of synthetic circRNAs Meganck, RM, et al.Molecular Therapy - Nucleic Acids Journal1/16/21336142328Asokan
Tools for experimental and computational analyses of off-target editing by programmable nucleasesBao, X Robert, et al.Nature Protocols1/16/2133288953Bao
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscleChen Z, et al.Science Advances1/7/2133523949Gersbach, Bursac, Truskey
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.Kloban, AL, et al.Nature1/6/2133408413Liu
Overcoming innate immune barriers that impede AAV gene therapy vectors Muhuri, M, et al.The Journal of Clinical Investigation1/4/2133393506Gao, Anderson, Xue
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue ConstructsHeifer, Abbigail, et al.Methods in Molecular Biology1/1/2132857373Gersbach, Bursac, Truskey
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical ApplicationsHu H, et al.Mater Today (Kidlington)1/1/2134421329Leong
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial functionJiang, Tingting, et al.Hepatology12/17/2033336367Gao, Anderson, Xue
Multicellular Systems to Translate Somatic Cell Genome Editors to Humans Hernandez-Gordillo V, et al.Current Opinion in Biotechnology12/16/2033718690Kiana
The dawn of non-human primate models for neurodevelopmental disorders.Aida, Tomoni, et al..Current Opinion in Genetics and Development12/1/2033718690Feng
Advances in microphysiological blood-brain barrier (BBB) models towards drug deliveryLee, Caleb S, et al.Current Opinion in Biotechnology12/1/2032711361Leong
Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation ScreensBlack, Joshua B, et al.Cell Reports12/1/2033264623Gersbach
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis.Leclair, Nathan K, et al.Molecular Cell11/19/2033176162Hinson
Phage-assisted continuous and non-continuous evolutionMiller, Shannon M, et al.Nature Protocols11/16/2033199872Liu
The once and future gene therapyBulaklak, Karen, et al.Nature Communications11/16/2033199717Gersbach, Bursac, Truskey | Gersbach
Delayed urea differential enhancement CEST (dudeCEST)-MRI with T 1 correction for monitoring renal urea handlingShin, Soo Hyun, et al.Magnetic Resonance in Medicine11/12/2033180343Vandsberger
Advances in Alpha-1 Antitrypsin Gene TherapyLorincz, Reka, et al.America Journal of Respiratory Cell and Nolecular Biology11/1/2032668173Curiel
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.Madigan, Victoria J, et al. Journal of Virology10/14/2032817219Asokan
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver Gong, Jing, et al.Advanced Materials10/14/2033053221Leong
3D kidney organoids for bench-to-bedside translationGupta, Navin, et al.Journal of Molecular Medicine10/9/2033034708Morizane, Lewis, Sabbisetti
Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV VectorsIbraheim, Raed, et al.bioRxiv10/9/20PreprintSontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 VariantsPettinato, Anthony M, et al.Circulation10/7/2033025817Hinson
Opportunities and limitations of genetically modified nonhuman primate models for neuroscience researchFeng, Guoping, et al.Proceeding of the National Academy of Sciences in the United States of America9/29/2032817435Feng
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular DystrophyKwon, Jennifer B, et al.Molecular Therapy Methods & Clinical Development9/28/2033145368Asokan | Gersbach, Bursac, Truskey
COVID-19 CG:Tracking SARS-CoV-2 mutations by locations and dates of interestChen, Albert Tian, et al.bioRxiv9/28/2032995794Deverman
Synthetic immunomodulation with a CRISPR super-repressor in vivoMoghadam, Farzaneh, et al.Nature Cell Biology9/22/2032884147Kiani
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided ApproachHavalik, Patrick L, et al.Journal of Virology9/15/2032669336Asokan
SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patientsPérez-Bermejo, Juan A, et al.bioRxiv9/12/2032884147McDevitt
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In VivoZhang, Kun, et al.Molecular Therapy Methods & Clinical Development9/11/2032953927Chen, Liu, Xu
Rapid, precise quantification of large DNA excisions and inversions by ddPCR.Watry, Hannah L, et al.Scientific Reports9/10/2032913194McDevitt
New Directions in Pulmonary Gene TherapyVu, A et al.Human Gene Therapy9/3/203281445McCray
A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries. Wang, Yuyuan, et al.Journal of Controlled Release8/10/2032380204Gong, Emborg, Levine, Roy, Saha
A scoutRNA Is Required for Some Type V CRISPR-Cas SystemsHarrington, Lucas B, et al.Molecular Cell8/6/2032645367Doudna, Banfield
NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer.Huang, Tung-Yung, et al.Cells8/3/2032756527Lam, Cheng
Finding order in slow axonal transportWang, Yuyuan, et al.Current Opinion in Neurobiology8/1/2032361600Gong, Emborg, Levine, Roy, Saha
DNA capture by a CRISPR-Cas9-guided adenine base editor.Al-Shayeb, Basem, et al.Science7/31/2032732424Doudna, Banfield | Liu
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury.Ettou, Sandrine, et al.Science Advances7/24/2032754639Morizane, Lewis, Sabbisetti
Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning.Arbab, Mandana, et al.Cell7/23/2032533916Liu
CRISPR-CasΦ from huge phages is a hypercompact genome editor.Pausch, Patrick, et al.Science7/17/2032675376Doudna, Banfield
Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. Li, Yamin, et al. Bioconjugate Chemistry7/15/2032520527Chen, Liu, Xu
Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers.Khodabukus, Alastair, et al.Toxicological Sciences7/1/2032294208Gersbach, Bursac, Truskey
The delivery challenge: fulfilling the promise of therapeutic genome editingvan Haasteren, Joost, et al.Nature Biotechnology6/29/2032601435Bankiewicz, Murthy
Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase.Wilson, Christopher, et al.Nature Biotechnology6/29/2032601430Liu
Polymeric vehicles for nucleic acid delivery.Piotrowski-Daspit, Alexandra S, et al.Advance Drug Delivery Reviews6/23/2032585159Saltzman, Glazer
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Anzalone, Andrew V, et al.Nature Biotechnology6/22/2032572269Liu
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing.Mok, Beverly Y, et al.Nature6/22/2032641830Liu
CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity.Lazzarotto, Cicera R, et al.Nature Biotechnology6/15/2032541958Tsai
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells.Nagpal, Neha, et al.Cell Stem Cell6/4/2032320679Sontheimer, Khvorova, Watts, Wolfe
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.Li, Ang, et al.Molecular Therapy6/3/2032348718Bao, Lagor
Can we mimic skeletal muscles for novel drug discovery?Broer, Torie, et al.Expert Opinion on Drug Delivery6/1/2032122176Gersbach, Bursac, Truskey
Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligandsGan Z, et al.Bioengineering & Translational Medicine5/27/2033758781Dahlman, Santangelo
YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells.Smith, Jordan L, et al.Hepatology5/26/2032452550Gao, Anderson, Xue | Liu
The developing toolkit of continuous directed evolutionMorrison, Mary S, et al.Nature Chemical Biology5/22/2032444838Liu
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct VectorsRumachik, Neil G, et al.Molecular Therapy, Methods & Clinical Development5/22/2032995354McDevitt
Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. Li, Yamin, et al.Angewandte Chemie International Edition in English5/21/2032438474Chen, Liu, Xu
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional ActivatorsKwon, Jennifer B, et al.Stem Cell Reports5/12/2032330446Gersbach, Bursac, Truskey | Asokan | Gersbach
Advances in genome editing for genetic hearing loss.Ding, Ning, et al.Advance Drug Delivery Reviews5/7/2032387678Bao, Lagor
Once upon a dish: engineering multicellular systemsHaase, Kristina, et al.Development5/4/2032366376Freedman
Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulationSeo, Jai Woong, et al.Nature Communications4/30/2032355221Lam, Cheng
Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope.Jiang, Tingting, et al.Nature Communications4/24/2032332735Gao, Anderson, Xue | Liu
Prime genome editing in rice and wheatLin, Qiupeng, et al.Nature Biotechnology3/16/2032393904Liu
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity.Richter, Michelle F, et al.Nature Biotechnology3/16/2032433547Liu
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic DiseasesWu, Shao-Shuai, et al.Theranostics3/15/2032292501Gao, Anderson, Xue
Targeted Delivery of Secretory Promelittin via Novel Poly(lactone‐co‐β‐amino ester) Nanoparticles for Treatment of Breast Cancer Brain MetastasesZhou, Yu, et al.Advanced Science3/7/2032154067Zhou
Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics.Liu, Esther, et al.Kidney3603/1/2032656538Freedman
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applicationsLi, Yamin, et al.Biomaterials3/1/2031945616Chen, Liu, Xu
Adenine base editing in an adult mouse model of tyrosinaemia.Song, Chun-Qing, et al.Nature Biomedical Engineering2/25/2031740768Liu | Gao, Anderson, Xue
Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo.Oyaghire, Stanley N, et al.Methods in Molecular Biology2/23/2032088877Saltzman, Glazer
High-throughput quantitative microscopy-based half-life measurements of intravenously injected agentsBracaglia, Laura G, et al.Proceeding of the National Academy of Sciences in the United States of America2/18/2032015123Saltzman, Glazer
Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine ZippersThadani, Nicole N, et al.ACS Synthetic Biology2/18/2032068391Bao, Lagor
Clades of huge phages from across Earth's ecosystemsAl-Shayeb, Basem, et al.Nature2/12/2032051592Doudna, Banfield
Quantitating Endosomal Escape of a Library of Polymers for mRNA DeliveryJiang, Yuhang, Lu, et al.Nano Letters2/12/2032003222Saltzman, Glazer
Continuous evolution of SpCas9 variants compatible with non-G PAMs.Miller, Shannon M, et al.Nature Biotechnology2/10/2032042170Liu
Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors.Doman, Jordan L, et al.Nature Biotechnology2/10/2032042165Liu
Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair.Economos, Nicholas G, et al.Molecules2/8/2032046275Saltzman, Glazer | Glazer, Ly, Saltzman
Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs.Osborn, Mark J, et al.The Journal of Investigative Dermatology2/1/2031437443Liu
From GWAS Association to Function: Candidate Gene Screening Within Insulin Resistance-Associated Genomic Loci Using a Preadipocyte Differentiation ModelLadha, Feria A, et al.Circulation Research1/31/2031999535Hinson
Detection of Extracellular Vesicle RNA Using Molecular Beacons.Oliveira Jr, Getulio Pereira de, et al.iScience1/24/2031958756Ghiran
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. Levy, JM, et al.Nature Biomedical Engineering1/4/2031937940Liu | Chen, Liu, Xu
Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings.Maynard, Lucy H, et al.Human Gene Therapy Methods12/19/1931855083McDevitt
Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States.Sun, W, et al.Molecular Cell12/19/1931668930Sontheimer, Khvorova, Watts, Wolfe
Optimizing biodegradable nanoparticle size for tissue-specific delivery.Mandl, HK, et al.Journal of Controlled Release11/28/1931654688Saltzman, Glazer
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsidsHuang, Qin, et al.PLOS ONE11/14/1931725765Deverman
Genome editing of HBG1 and HBG2 to induce fetal hemoglobinMétais, Jean-Yves, et al.Blood Advances11/12/1931698466Tsai
Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epitheliaSateesh Krishnamurthy, et al.Nature Communications10/28/1931659165McCray
Search-and-replace genome editing without double-strand breaks or donor DNAAnzalone AV, et al.Nature10/21/1931634902Liu
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo.Ibraheim, Raed, et al.Genome Biology9/19/1930231914Gao, Anderson, Xue
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editingChen G, et al.Nature Nanotechnology9/9/1931501532Gong, Emborg, Levine, Roy, Saha
Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?Qiu, Min, et al.Biomacromolecules9/9/1931342740Chen, Liu, Xu
High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs.Xu, Meiyu, et al.Human Gene Therapy8/30/1931072208Gao, Anderson, Xue
Mapping cis-regulatory chromatin contacts in neural cells links neuropsychiatric disorder risk variants to target genes.Song, Michael, et al.Nature Genetics7/31/1931367015McDevitt
Continuous evolution of base editors with expanded target compatibility and improved activityThuronyi BW, et al.Nature Biotechnology7/22/1931332326Liu
Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis.Yang, Yeon-Suk, et al.Nature Communications7/4/1931273195Gao, Anderson, Xue
An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cellsKim, Y Bill, et al.Nature Communications7/2/1931266953Liu
Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles.Liu, J, et al.Advanced Materials6/19/1931215123Chen, Liu, Xu
Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes.Kwan, Suet-Yan, et al.Hepatology6/12/1931188495Gao, Anderson, Xue
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors.Huang, Tony P, et al.Nature Biotechnology6/10/1931110355Liu
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunityXiao, Yuanyuan, et al.JCI Insight5/21/1931112525Gao, Anderson, Xue
Adeno-associated virus vector as a platform for gene therapy delivery.Wang D, et al.Nature Reviews Drug Discovery5/18/1930710128Gao, Anderson, Xue
Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks.Rees, Holly A, et al.Nature Communications5/17/1931101808Liu
Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for AdrenomyeloneuropathyGong, Yi, et al.Human Gene Therapy5/15/1930358470Gao, Anderson, Xue
Analysis and minimization of cellular RNA editing by DNA adenine base editorsRees HA, et al.Science Advances5/8/1931086823Liu
Nucleic Acid Therapeutics for Neurological DiseasesWatts, Jonathan K, et al.Neurotherapeutics4/29/1931037650Sontheimer, Khvorova, Watts, Wolfe
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-SeqWienert, Beeke, et al.Science4/19/1931000663McDevitt
CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling.Mou, Haiwei, et al.Genome Medicine4/16/1930987660Gao, Anderson, Xue
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging.Oh, Younghee, et al.Nature Chemical Biology4/1/1930936501McDevitt
Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression.Martinez-Navio, et al.Immunity3/19/1930850342Gao, Anderson, Xue
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing AntibodiesGardner, Matthew R, et al.Molecular Therapy3/6/1930704961Gao, Anderson, Xue
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome EditingEdraki, Alireza, et al.Molecular Cell2/21/1930581144Gao, Anderson, Xue
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems.Wang, Yuyuan, et al.Bioconjugate Chemistry2/20/1930592619Gong, Emborg, Levine, Roy, Saha
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous SystemGessler, Dominic J, et al.Methods in Molecular Biology2/20/1930783972Gao, Anderson, Xue
Quantitative and Digital Droplet-Based AAV Genome TitrationSanmiguel, Julio, et al.Methods in Molecular Biology2/20/1930783968Gao, Anderson, Xue
Simultaneous targeting of linked loci in mouse embryos using base editingLee, Hye Kyung, et al.Scientific Reports2/7/1930733567Liu
Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome EditingChang, Jin, et al.Accounts of Chemical Research12/26/1830586281Chen, Liu, Xu
Illuminating the genome-wide activity of genome editors for safe and effective therapeuticsCheng, Yong, et al.Genome Biology12/22/1830577870Tsai
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene TransferWang, Dan, et al.Molecular Therapy: Methods & Clinical Development12/14/1830397628Gao, Anderson, Xue
MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7Luo, Xufeng, et al.Molecular Therapy: Methods & Clinical Development12/14/1830406154Gao, Anderson, Xue
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid NanoparticlesYamin Li, et al.Advanced Healthcare Materials11/22/1830565897Chen, Liu, Xu
Targeting fidelity of adenine and cytosine base editors in mouse embryosLee, Hye Kyung, et al.Nature Communications11/15/1830442934Liu
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene TherapyLiang, Xiao, et al.Human Gene Therapy11/13/1829893153Gao, Anderson, Xue
Understanding and repurposing CRISPR-mediated alternative splicingSmith, Jordan L, et al.Genome Biology11/6/1830400804Gao, Anderson, Xue
Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq.Lazzarotto, Cicera R, et al.Nature Protocols10/19/1830341435Tsai
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