|
| 38033325 | Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. | Nucleic Acids Res | 2024 |
| 36993169 | Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing <i>in vivo</i>. | bioRxiv | 2023 |
| 36424489 | Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases. | Nat Biotechnol | 2023 |
| 36184851 | WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects. | Molecular Therapy | 2023 |
| 37547286 | Therapeutic inhibition of miR-155 attenuates liver fibrosis via STAT3 signaling. | | 2023 |
| 37153616 | A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors. | | 2023 |
| 37131611 | Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope. | | 2023 |
| 36996170 | Base editing rescue of spinal muscular atrophy in cells and in mice. | | 2023 |
| 36933560 | Learning to lock down genetic diseases without throwing away the key. | | 2023 |
| 35322228 | AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. | Nature | 2022 |
| 36369293 | Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration. | Nat Commun | 2022 |
| 36178359 | Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. | Human Gene Therapy | 2022 |
| 35950212 | AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy. | Molecular Therapy - Nucleic Acids | 2022 |
| 35811581 | Adenine Base Editing with a Single Adeno-Associated Virus Vector. | | 2022 |
| 35988648 | Paradoxical activation of transcription factor SREBP1c and de novo lipogenesis by hepatocyte-selective ATP-citrate lyase depletion in obese mice. | J Biol Chem | 2022 |
| 36258013 | Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery. | Nat Commun | 2022 |
| 35169297 | Regulation of sclerostin by the SIRT1 stabilization pathway in osteocytes. | Cell Death and Differentiation | 2022 |
| 34794463 | Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein-Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBVâ¿¿+â¿¿lymphoproliferative disorders. | Virology Journal | 2021 |
| 34182803 | Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis. | Hum Gene Ther | 2021 |
| 33568518 | Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses. | Sci Transl Med | 2021 |
| 34338329 | Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency. | Annals of Neurology | 2021 |
| 34631930 | Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette. | Molecular Therapy - Methods and Clinical Development | 2021 |
| 31277075 | Conditional, inducible gene silencing in dopamine neurons reveals a sex-specific role for Rit2 GTPase in acute cocaine response and striatal function. | | 2020 |
| 32015438 | LATS suppresses mTORC1 activity to directly coordinate Hippo and mTORC1 pathways in growth control. | Nature Cell Biology | 2020 |
| 31843447 | Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold. | Molecular Therapy | 2020 |
| 32405514 | Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy. | Molecular Therapy - Methods and Clinical Development | 2020 |
| 31826994 | Comparative Analysis of the Capsid Structures of AAVrh.10, AAVrh.39, and AAV8. | Journal of Virology | 2020 |
| 32100151 | Patient-derived xenograft models for personalized medicine in colorectal cancer. | Clinical and Experimental Medicine | 2020 |
| 32775498 | AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity. | Molecular Therapy - Methods and Clinical Development | 2020 |
| 31072208 | High-Throughput Quantification of Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. | Human Gene Therapy | 2019 |
| 31824476 | Inhibition of miR-378a-3p by Inflammation Enhances IL-33 Levels: A Novel Mechanism of Alarmin Modulation in Ulcerative Colitis. | Frontiers in Immunology | 2019 |
| 31273195 | Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. | Nature Communications | 2019 |
| 29554131 | Down-regulation of microRNA-203-3p initiates type 2 pathology during schistosome infection via elevation of interleukin-33. | PLoS Pathogens | 2018 |
| 28987423 | MicroRNA 122, Regulated by GRLH2, Protects Livers of Mice and Patients From Ethanol-Induced Liver Disease. | Gastroenterology | 2018 |
| 30406154 | Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing . | Molecular Therapy - Methods and Clinical Development | 2018 |
| 30397628 | Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. | Molecular Therapy - Methods and Clinical Development | 2018 |
| 29766031 | A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates. | Molecular Therapy - Methods and Clinical Development | 2018 |
| 29766023 | Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras. | Molecular Therapy - Methods and Clinical Development | 2018 |
| 28462820 | Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity. | Molecular Therapy | 2017 |
| 25546547 | Recombinant adeno-associated virus-mediated inhibition of microRNA-21 protects mice against the lethal schistosome infection by repressing both IL-13 and transforming growth factor beta 1 pathways. | Hepatology | 2015 |
| 25632938 | Adeno-associated virus-mediated microRNA delivery and therapeutics. | Seminars in Liver Disease | 2015 |
| 23621841 | Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. | Hum Gene Ther | 2013 |
| 23817205 | A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. | Molecular Therapy | 2013 |
| 24010702 | Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer. | Human Gene Therapy | 2013 |
| 22388288 | Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. | Nat Methods | 2012 |
| 21179009 | MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. | Mol Ther | 2011 |
| 19618999 | Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production. | Human Gene Therapy | 2009 |