Molecular Therapy : the Journal of the American Society of Gene Therapy
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Publication Venue For
- AAV-CNS matters turn from gray to white. 2021
- The Path to Progress Preclinical Studies of Age-Related Neurodegenerative Diseases: A Perspective on Rodent and hiPSC-Derived Models.. 29:949-972. 2021
- Controlling cancer-induced inflammation with a nucleic acid scavenger prevents lung metastasis in murine models of breast cancer. 2020
- In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.. 28:1931. 2020
- Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.. 28:1455-1463. 2020
- Preclinical Development of a vWF Aptamer to Limit Thrombosis and Engender Arterial Recanalization of Occluded Vessels.. 27:1228-1241. 2019
- Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD.. 26:2638-2649. 2018
- Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease.. 26:2304-2314. 2018
- In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.. 26:1818-1827. 2018
- Therapeutic Benefit of Autophagy Modulation in Pompe Disease.. 26:1783-1796. 2018
- In Vivo Expansion and Antitumor Activity of Coinfused CD28- and 4-1BB-Engineered CAR-T Cells in Patients with B Cell Leukemia.. 26:976-985. 2018
- Polymer-Mediated Inhibition of Pro-invasive Nucleic Acid DAMPs and Microvesicles Limits Pancreatic Cancer Metastasis.. 26:1020-1031. 2018
- Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier.. 26:510-523. 2018
- Differential Induction of Immunogenic Cell Death and Interferon Expression in Cancer Cells by Structured ssRNAs.. 25:1295-1305. 2017
- Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.. 25:1199-1208. 2017
- Involvement of a Rac1-Dependent Macropinocytosis Pathway in Plasmid DNA Delivery by Electrotransfection.. 25:803-815. 2017
- Immunization with an SIV-based IDLV Expressing HIV-1 Env 1086 Clade C Elicits Durable Humoral and Cellular Responses in Rhesus Macaques.. 24:2021-2032. 2016
- In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.. 24:697-706. 2016
- Smooth Muscle Cell-targeted RNA Aptamer Inhibits Neointimal Formation.. 24:779-787. 2016
- Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.. 24:53-65. 2016
- Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.. 23:523-532. 2015
- Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.. 22:2038-2045. 2014
- Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.. 22:702-712. 2014
- Enhanced oncolytic virotherapy through oxidative stress inhibition.. 21:1981-1983. 2013
- Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.. 21:1661-1667. 2013
- Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.. 21:18-30. 2013
- The AAV vector toolkit: poised at the clinical crossroads.. 20:699-708. 2012
- Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.. 20:443-455. 2012
- Rapidly regulating platelet activity in vivo with an antidote controlled platelet inhibitor.. 20:391-397. 2012
- Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.. 19:1961-1970. 2011
- Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.. 19:1070-1078. 2011
- MAPK signal-integrating kinase controls cap-independent translation and cell type-specific cytotoxicity of an oncolytic poliovirus.. 18:1937-1946. 2010
- Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.. 18:353-360. 2010
- Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo.. 17:343-351. 2009
- RNA aptamer blockade of osteopontin inhibits growth and metastasis of MDA-MB231 breast cancer cells.. 17:153-161. 2009
- Recombinant oncolytic poliovirus eliminates glioma in vivo without genetic adaptation to a pathogenic phenotype.. 16:1865-1872. 2008
- Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.. 16:1366-1371. 2008
- An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.. 16:1316-1322. 2008
- Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.. 16:1252-1260. 2008
- AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.. 16:665-672. 2008
- In vivo reprogramming of hTERT by trans-splicing ribozyme to target tumor cells.. 16:74-80. 2008
- RNA Aptamer-targeted Inhibition of NF-κB Suppresses Non-small Cell Lung Cancer Resistance to Doxorubicin.. 16:66-73. 2008
- Corrigendum to "Efficacy of Helper-dependent Adenovirus Vector-mediated Gene Therapy in Murine Glycogen Storage Disease Type Ia".. 15:2051. 2007
- Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.. 15:1253-1258. 2007
- In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using non-primate lentiviral vectors. 3:850-856. 2001