Publication TitleAuthorsJournalPub DatePubMedProject PIs
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
COVID-19 CG:Tracking SARS-CoV-2 mutations by locations and dates of interestChen, Albert Tian, et al.bioRxiv 9/28/2032995794Deverman
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
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 Systems.Harrington, Lucas B, et al.Molecular Cell8/6/2032645367Doudna, Banfield
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
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, 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
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
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.Proc Natl Acad Sci U S A2/18/2032015123Saltzman, 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
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 Science1/19/2032154067Zhou
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, Song, Chun-Qing, Mir, Aamir, 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, Berenson, Anna, Laheji, Fiza, et al.Human Gene Therapy5/15/1930358470Gao, Anderson, Xue
Analysis and minimization of cellular RNA editing by DNA adenine base editorsRees HA, Wilson C, Doman JL, Liu DRScience Advances5/8/1931086823Lui
Nucleic Acid Therapeutics for Neurological DiseasesWatts, Jonathan K, Brown, Robert H, Khvorova, AnastasiaNeurotherapeutics4/29/1931037650Sontheimer, 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