Publication TitleAuthorsJournalPub DatePubMedProject PIs
Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routesChen, CC, et al.Pharmaceutical Patent Analysis33467938Chen, Liu, Xu
Precision genome editing using cytosine and adenine base editors in mammalian cells Huang, TP, et al.Nature Protocols1/18/2133462442Lui
Tools for experimental and computational analyses of off-target editing by programmable nucleasesBao, X Robert, et al.Nature Protocols1/16/2133288953Bao
In vivo base editing rescues Hutchinson–Gilford progeria syndrome in miceKloban, Luke W, et al.Nature1/6/2133408413Lui
Overcoming innate immune barriers that impede AAV gene therapy vectors Muhuri, M, et al.The Journal of Clinical Investigation1/4/2133393506Gao
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue ConstructsHeifer, Abbigail, et al.Methods in Molecular Biology1/1/2132857373Gersbach, Bursac, Truskey
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
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
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
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 | Lui
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.Technological 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
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 | Lui
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, Eng.5/21/2032438474Chen, 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 | Lui
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9Li, Ang, Tanner, et al.Molecular Therapy4/19/2032348718Heaney, Dickinson, Lagor | Bao, Lagor
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 Mol Biol2/23/2032088877Saltzman, 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 Dermatology2/1/2031437443Lui
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.Nature10/21/1931634902Lui
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 Biotechnology7/22/1931332326Lui
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 Communications7/2/1931266953Lui
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 Biotechnology6/10/1931110355Lui
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 Communications5/17/1931101808Lui
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 Advances5/8/1931086823Lui
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 Mol Biol2/20/1930783972Gao, Anderson, Xue
Quantitative and Digital Droplet-Based AAV Genome TitrationSanmiguel, Julio, et al.Methods in Mol Biol2/20/1930783968Gao, Anderson, Xue
Simultaneous targeting of linked loci in mouse embryos using base editingLee, Hye Kyung, et al.Scientific Reports2/7/1930733567Lui
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 Communications11/15/1830442934Lui
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
Cas9-mediated Allelic Exchange Repairs Compound Heterozygous Recessive Mutations in MiceWang, Dan, et al.Nature Biotechnology10/1/1830102296Gao, Anderson, Xue
The temporal landscape of recursive splicing during Pol II transcription elongation in human cellsZhang, Xiao-Ou, et al.Plos Genetics8/27/1830148885Gao, Anderson, Xue