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| 37526463 | Evaluating Novel Protein Phosphatase 2A Activators as Therapeutics for Emphysema. | Am J Respir Cell Mol Biol | 2023 |
| 35121111 | Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency. | Molecular Therapy | 2022 |
| 35592360 | Liver-directed gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice. | Molecular Therapy - Methods and Clinical Development | 2022 |
| 35508619 | Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates. | Nat Commun | 2022 |
| 36271076 | CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro. | Nat Commun | 2022 |
| 35145305 | AAV gene therapy for Tay-Sachs disease. | Nat Med | 2022 |
| 35104244 | Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease. | JCI insight | 2022 |
| 34724811 | SepA Enhances Invasion of Epithelial Cells by Degrading Alpha-1 Antitrypsin and Producing a Neutrophil Chemoattractant. | mBio | 2021 |
| 33892050 | Genetic diversity of axon degenerative mechanisms in models of Parkinson's disease. | Neurobiology of Disease | 2021 |
| 33554955 | Alpha-1 antitrypsin deficiency impairs lung antibacterial immunity in mice. | JCI insight | 2021 |
| 34244428 | Hypoimmune induced pluripotent stem cell-derived cell therapeutics treat cardiovascular and pulmonary diseases in immunocompetent allogeneic mice. | Proceedings of the National Academy of Sciences of the United States of America | 2021 |
| 33649241 | Up-regulation of miR-34b/c by JNK and FOXO3 protects from liver fibrosis. | Proceedings of the National Academy of Sciences of the United States of America | 2021 |
| 32858321 | A Translational Model for Venous Thromboembolism: MicroRNA Expression in Hibernating Black Bears. | J Surg Res | 2021 |
| 32498636 | Intralingual Administration of AAVrh10-miR Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis. | Human Gene Therapy | 2020 |
| 31970202 | Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model. | Molecular Therapy - Methods and Clinical Development | 2020 |
| 32093728 | Dipeptide repeat proteins inhibit homology-directed DNA double strand break repair in C9ORF72 ALS/FTD. | Molecular Neurodegeneration | 2020 |
| 31982038 | Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans. | Molecular Therapy | 2020 |
| 32640133 | <i>SOD1</i> Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS. | N Engl J Med | 2020 |
| 32723872 | CHOP and c-JUN up-regulate the mutant Z α-antitrypsin, exacerbating its aggregation and liver proteotoxicity. | Journal of Biological Chemistry | 2020 |
| 32607885 | Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. | Methods in Molecular Biology | 2020 |
| 30706405 | Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons. | Methods in Molecular Biology | 2019 |
| 30383447 | Lack of acute xenogeneic graft- versus-host disease, but retention of T-cell function following engraftment of human peripheral blood mononuclear cells in NSG mice deficient in MHC class I and II expression. | FASEB J | 2019 |
| 31070450 | Genetic Deficiency of Flavin-Containing Monooxygenase 3 ( Fmo3) Protects Against Thrombosis but Has Only a Minor Effect on Plasma Lipid Levels-Brief Report. | Arteriosclerosis, Thrombosis, and Vascular Biology | 2019 |
| 31112525 | Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. | JCI Insight | 2019 |
| 30783965 | Design of AAV Vectors for Delivery of RNAi. | Methods in Molecular Biology | 2019 |
| 30944467 | Precise therapeutic gene correction by a simple nuclease-induced double-stranded break. | Nature | 2019 |
| 30828586 | Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study. | Molecular Therapy - Methods and Clinical Development | 2019 |
| 31375812 | A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system. | Nat Biotechnol | 2019 |
| 29453277 | Editing out five paralogs to create a mouse model of genetic emphysema. | Proceedings of the National Academy of Sciences of the United States of America | 2018 |
| 28826231 | Gene Therapy with an Adeno-Associated Viral Vector Expressing Human Interleukin-2 Alters Immune System Homeostasis in Humanized Mice. | Hum Gene Ther | 2018 |
| 30381409 | Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques. | Science Translational Medicine | 2018 |
| 30320596 | Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. | Journal of Huntington's disease | 2018 |
| 29207890 | Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease. | Human Gene Therapy | 2018 |
| 29597895 | In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. | Hum Gene Ther | 2018 |
| 28095479 | Central Role of IL-23 and IL-17 Producing Eosinophils as Immunomodulatory Effector Cells in Acute Pulmonary Aspergillosis and Allergic Asthma. | PLoS Pathogens | 2017 |
| 28771235 | Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques. | Gene Therapy | 2017 |
| 28753427 | A Genetic Variant Associated with Five Vascular Diseases Is a Distal Regulator of Endothelin-1 Gene Expression. | Cell | 2017 |
| 28624208 | Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin. | Molecular Therapy - Nucleic Acids | 2017 |
| 28752462 | Quantification of Z-AAT by a Z-Specific "Sandwich" ELISA. | Methods in Molecular Biology | 2017 |
| 28752461 | Quantification of Murine AAT by Direct ELISA. | Methods in Molecular Biology | 2017 |
| 28752450 | Design, Cloning, and In Vitro Screening of Artificial miRNAs to Silence Alpha-1 Antitrypsin. | Methods in Molecular Biology | 2017 |
| 28408179 | 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. | Molecular Therapy | 2017 |
| 28323492 | Regulatory and Exhausted T Cell Responses to AAV Capsid. | Human Gene Therapy | 2017 |
| 29032169 | Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency. | Mol Ther | 2017 |
| 28606110 | A C9ORF72 BAC mouse model recapitulates key epigenetic perturbations of ALS/FTD. | Molecular Neurodegeneration | 2017 |
| 28137880 | Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency. | Proceedings of the National Academy of Sciences of the United States of America | 2017 |
| 26710998 | Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. | Human Gene Therapy | 2016 |
| 27023329 | Self-Complementary Adeno-Associated Virus Vectors Improve Transduction Efficiency of Corneal Endothelial Cells. | PLoS ONE | 2016 |
| 26891182 | Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. | Annals of Neurology | 2016 |
| 27003665 | Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon? | Journal of Huntington's disease | 2016 |