Molecular Therapy

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  Publication Venue For

  • Preclinical Development of a vWF Aptamer to Limit Thrombosis and Engender Arterial Recanalization of Occluded Vessels. 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
  • It's All in the Delivery: Designing Hydrogels for Cell and Non-viral Gene Therapies..  26:2087-2106. 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
  • Boosting, Not Breaking: CRISPR Activators Treat Disease Models..  26:334-336. 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
  • 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
  • Smooth Muscle Cell-targeted RNA Aptamer Inhibits Neointimal Formation..  24:779-787. 2016
  • Genome-editing Technologies for Gene and Cell Therapy..  24:430-446. 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
  • 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
  • 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
  • Erratum: Reading Frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients (Molecular Therapy (2013) 21 (1718-1726) DOI: 10.1038/mt.2013.111).  21:2130. 2013
  • Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease..  21:1661-1667. 2013
  • Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients..  21:1718-1726. 2013
  • Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases..  21:266-268. 2013
  • Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.  21:18-30. 2013
  • Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice..  21:18-30. 2013
  • Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients.  21:1718-1726. 2013
  • A tubulin binding peptide targets glioma cells disrupting their microtubules, blocking migration, and inducing apoptosis..  20:1367-1377. 2012
  • Isolation and optimization of murine IL-10 receptor blocking oligonucleotide aptamers using high-throughput sequencing..  20:1242-1250. 2012
  • The AAV vector toolkit: poised at the clinical crossroads..  20:699-708. 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
  • Targeting 4-1BB costimulation to disseminated tumor lesions with bi-specific oligonucleotide aptamers..  19:1878-1886. 2011
  • Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer..  19:1070-1078. 2011
  • Targeting tumor vasculature with an oncolytic virus..  19:886-894. 2011
  • Improving adoptive T cell therapy by targeting and controlling IL-12 expression to the tumor environment..  19:751-759. 2011
  • MAPK signal-integrating kinase controls cap-independent translation and cell type-specific cytotoxicity of an oncolytic poliovirus..  18:1937-1946. 2010
  • Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo..  18:812-818. 2010
  • Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease..  18:353-360. 2010
  • Engineering clustered ligand binding into nonviral vectors: alphavbeta3 targeting as an example..  17:828-836. 2009
  • 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
  • A critical role for type I IFN-dependent NK cell activation in innate immune elimination of adenoviral vectors in vivo..  16:1300-1307. 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
  • 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
  • Quantitative comparison of intracellular unpacking kinetics of polyplexes by a model constructed from quantum dot-FRET..  16:324-332. 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
  • RNA aptamer-targeted inhibition of NF-kappa B 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
  • Successful immunization with a single injection of non-integrating lentiviral vector.  15:1716-1723. 2007
  • Successful immunization with a single injection of non-integrating lentiviral vector..  15:1716-1723. 2007
  • Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia..  15:1253-1258. 2007
  • Effects of MIP-1 alpha, MIP-3 alpha, and MIP-3 beta on the induction of HIV Gag-specific immune response with DNA vaccines..  15:1007-1015. 2007
  • Biomaterials approach to expand and direct differentiation of stem cells..  15:467-480. 2007
  • Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II..  14:822-830. 2006
  • Mesenchymal stem cells overexpressing Akt dramatically repair infarcted myocardium and improve cardiac function despite infrequent cellular fusion or differentiation..  14:840-850. 2006
  • Multiple innate inflammatory responses induced after systemic adenovirus vector delivery depend on a functional complement system..  14:588-598. 2006
  • A novel antidote-controlled anticoagulant reduces thrombin generation and inflammation and improves cardiac function in cardiopulmonary bypass surgery..  14:408-415. 2006
  • Adeno-associated virus serotypes: vector toolkit for human gene therapy..  14:316-327. 2006
  • Polyclonal long-term MFGS-gp91phox marking in rhesus macaques after nonmyeloablative transplantation with transduced autologous peripheral blood progenitor cells..  14:202-211. 2006
  • Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice..  14:1-2. 2006
  • 219. Persistent Correction of Hyperphenylalaninemia Following Liver-Directed, rAAV2/8-Mediated Gene Therapy for Murine Phenylketonuria (PKU).  13:S84-S84. 2006
  • 427. Adenovirus Mediated GRP94/gp96 Expression in Treatment of Neuroblastoma.  13:S164-S164. 2006
  • 954. High Intensity Focused Ultrasound (HIFU) Induced Trans-Gene Activation in Solid Tumors.  13:S368-S368. 2006
  • Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice..  13:127-134. 2006
  • Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II..  12:876-884. 2005
  • Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter..  11:889-898. 2005
  • Retargeting mobile group II introns to repair mutant genes..  11:687-694. 2005
  • Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II..  11:57-65. 2005
  • Recombinant adenovirus vectors activate the alternative complement pathway, leading to the binding of human complement protein C3 independent of anti-ad antibodies..  10:1140-1142. 2004
  • Methotrexate and cytarabine inhibit progression of human lymphoma in NOD/SCID mice carrying a mutant dihydrofolate reductase and cytidine deaminase fusion gene..  10:574-584. 2004
  • Ribozyme-mediated induction of apoptosis in human cancer cells by targeted repair of mutant p53 RNA..  10:365-372. 2004
  • Uncertain benefit: investigators' views and communications in early phase gene transfer trials..  10:225-231. 2004
  • Transfection of RNA encoding tumor antigens following maturation of dendritic cells leads to prolonged presentation of antigen and the generation of high-affinity tumor-reactive cytotoxic T lymphocytes..  9:757-764. 2004
  • A quantitative method for measuring gene expression magnitude and volume delivered by gene therapy vectors..  9:625-631. 2004
  • HIV-1 Vif: counteracting innate antiretroviral defenses..  8:525-527. 2003
  • GENIS: gene expression of sodium iodide symporter for noninvasive imaging of gene therapy vectors and quantification of gene expression in vivo..  8:508-518. 2003
  • Catheter-based intracoronary myocardial adenoviral gene delivery: importance of intraluminal seal and infusion flow rate..  8:306-313. 2003
  • Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector..  7:467-477. 2003
  • Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector..  7:193-201. 2003
  • Noninvasive imaging of cationic lipid-mediated delivery of optical and PET reporter genes in living mice..  6:555-562. 2002
  • Optimizing aptamer activity for gene therapy applications using expression cassette SELEX..  6:30-34. 2002
  • Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model..  5:780-787. 2002
  • Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction..  5:436-446. 2002
  • Generation of species cross-reactive aptamers using "toggle" SELEX..  4:567-573. 2001
  • Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing..  4:105-114. 2001
  • Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach..  3:787-792. 2001
  • Transgene expression in the brain stem effected by intramuscular injection of polyethylenimine/DNA complexes..  3:658-664. 2001
  • Resistance of pancreatic carcinoma cells is reversed by coculturing NK-like T cells with dendritic cells pulsed with tumor-derived RNA and CA 19-9..  3:54-60. 2001
  • Combination treatment of murine tumors by adenovirus-mediated local B7/IL12 immunotherapy and radiotherapy..  2:195-203. 2000
  • Enhancing RNA repair efficiency by combining trans-splicing ribozymes that recognize different accessible sites on a target RNA..  2:245-255. 2000
  • Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors..  2:16-25. 2000
  • A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor 2019
  • AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy 2019
  • Correction of Glycogen Storage Disease Type III with an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme 2019
  • Design Principles for AAV Mediated Circular RNA Expression in the Brain 2019
  • Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution 2019
  • Inhalation of an RNA Aptamer Targeting Extracellular Histones Protects from Acute Lung Injury 2019
  • Integrase-Deficient Lentiviral Vector for Efficient CRISPR/Cas9-Mediated Gene and Epigenome-Editing Applications 2019
  • Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy 2019
  • Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription 2019
  • Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9 2019
  • Respiratory Directed Gene Therapy to Silence SOD1 Prolongs Survival in the SOD1 ALS Mouse 2019
  • Salmeterol with Liver Depot Gene Therapy Reversed Biochemical and Autophagic Abnormalities of Skeletal Muscle in Pompe Disease 2019
  • The New Epigenome-Editing Approach for Targeting Dysregulated SNCA Expression: Novel Target Validation for Next-Generation Drug Discovery 2019
  • Tumoricidal Stem Cell Therapy Enables Killing in Novel Hybrid Models of Heterogeneous Glioblastoma 2019
  • A Single Intravenous Injection of an AAV-PHP. B Vector Encoding Human Acid alpha-Glucosidase Corrects Both Muscle and Brain Defects in Murine Pompe Disease 2018
  • Bezafibrate Enhances AAV Gene Therapy in Glycogen Storage Disease Type Ia 2018
  • Capsid-Glycan Receptor Interactions Influence AAV Transport across the Blood-Brain Barrier 2018
  • Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9 2018
  • Expression of CRISPR-Cas9 Single Guide RNA's Using Small tRNA Promoters 2018
  • Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy 2018
  • Mapping and Engineering Functional Domains of the Assembly Activating Protein of Adeno-Associated Viruses 2018
  • Novel Heterogeneous Glioblastoma Models to Optimize Human Tumoricidal Stem Cell Therapy 2018
  • RNF121 is a Key Transcriptional Regulator of AAV Genome Expression 2018
  • Systemic Expression of Translatable Circular Rnas Using Recombinant AAV Vectors 2018
  • VSMC Specific Aptamer Ligands for the Next Generation of DES to Prevent Neointimal Formation 2018
  • Wide Spread Adenoviral Vector Delivery to a Cardiac Allograft Utilizing an Ex Vivo Perfusion Storage Strategy 2018
  • Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain Barrier 2017
  • Disruption of Water Homeostasis Affects AAV Transport Across the Blood-Brain Barrier 2017
  • Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9 2017
  • Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema 2017
  • Effect of AAV Gene Therapy on Hepatic Tumor Induced by Deficiency of Glucose-6-Phosphatase 2017
  • Expanding the CNS Transduction Profile of AAV4 by Structure-Guided Evolution 2017
  • Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease 2017
  • Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy 2017
  • Salmeterol, a Beta-Agonist, Enhances Therapeutic Efficacy in Pompe Gene Therapy 2017
  • Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape Variants 2017
  • Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene Transfer 2017
  • Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe Disease 2017
  • Treatment of Sepsis by Neutralization of Extracellular Histones with Nucleic Acid Aptamers 2017
  • 167. Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPD 2016
  • 258. Phase I/IIa Study of TT-034, a DNA-Directed RNA Interference (ddRNAi) Agent Delivered as a Single Administration for the Treatment of Subjects with Chronic Hepatitis C Virus (HCV) 2016
  • 311. In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Enhances Biochemical Correction in Mice with Glycogen Storage Disease Type IA 2016
  • 381. Prohypertrophic Agents Enhance the Response to Gene Therapy in Pompe Disease 2016
  • 482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy 2016
  • 49. Minimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior Estimates 2016
  • 504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy 2016
  • 727. Characterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopes 2016
  • 8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction 2016
  • 93. Development of AAVidua Vector for the Treatment of Cornea Associated Vision Loss in Bone Marrow Treated-Hurler Patients 2016
  • Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer 2016
  • 14. Next Generation AAV Vectors for Limiting Systemic Leakage and Improving Safety Following CNS Administration 2015
  • 265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain 2015
  • 272. Nucleic Acid Scavenging Nanofibers as Anti-Inflammatory Meshes 2015
  • 30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector 2015
  • 302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors 2015
  • 38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction 2015
  • 397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy 2015
  • 41. U2 snRNP Spliceosome Proteins Block Recombinant AAV Vector Transduction 2015
  • 42. Differential Transduction Profiles of AAV Vectors in a Mouse Model of Human Glycosylation 2015
  • 472. Low-Shear Sodding of an Implantable Expanded Polytetrafluoroethylene Scaffold with a Rat Adipose-Derived Stromal-Vascular Fraction 2015
  • 485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements 2015
  • 59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype 2015
  • 61. Vascular Smooth Muscle Cell RNA Aptamers for the Treatment of Cardiovascular Disease 2015
  • 66. Identification and Characterization of a B Cell Aptamer That Targets Diffuse Large B Cell Lymphoma (DLBCL) and Chronic Myelogenous Leukemia (CML) 2015
  • 701. Long-Term Complications of Glycogen Storage Disease Type IA in the Dog Model Treated With Gene Replacement Therapy 2015
  • A Novel Aptamer Targeting Agent for Prostate Cancer 2015
  • Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors 2015
  • Bioengineered Skeletal Muscle With Functional Stem Cell Pool and Capacity for Vascular Integration and Maturation In Vivo 2014
  • CRISPR/Cas9-Based Transcriptional Repressors for Control of Human Gene Expression 2014
  • Cell-Targeted RNA-Based Therapies for Cardiovascular Disease 2014
  • Characterizing Essential Receptor Binding Residues for AAV1 2014
  • Gene Therapy for Heart Disease Using Electrically Active Fibroblasts 2014
  • Human Pluripotent Stem Cell-Derived Cardiac Tissue Patch for Use in Cell-Based Cardiac Therapy 2014
  • Mapping the Antigenic Structure of Adeno-Associated Virus Serotypes 8 and 9 2014
  • Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods 2014
  • Neonatal rAAV2/8 Vector Administration Does Not Prevent Therapeutic Response To Repeat Vector Injection in Adult Pah(enu2/enu2) Mice 2014
  • Nucleic-Acid Binding Polymers as Anti-Inflammatory Agents 2014
  • Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) 2014
  • Regional-CNS MiniPromoters for AAV Are Identified in a High Through-Put Pipeline 2014
  • Tissue Engineered Human Skeletal Muscle as a Pre-Clinical Model for AAV Treatment of Pompe Disease 2014
  • Adjunctive beta 2-Agonist Therapy Enhances Trafficking of Acid alpha-Glucosidase to Lysosomes in the Skeletal Muscle of Mice with Pompe Disease 2013
  • Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy: Long-Term Outcome and Comparison of Efficacy with Allogeneic Hematopoietic Stem Cell Transplantation 2013
  • Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo 2013
  • Pathogenesis of Growth Failure in Murine and Canine Glycogen Storage Disease-Ia and Partial Reversal of Growth Failure Using Adeno-Associated Viral Vector Therapy 2013
  • Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4 2013
  • Sialic Acids and Erythrocyte Interactions Are Critical Determinants of Cardiopulmonary Tropism Displayed by AAV Serotype 4 2013
  • Smooth Muscle Cell Targeted RNA Aptamers for the Treatment of Vascular Disease 2013
  • Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors 2013
  • Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors 2012
  • Aptamer-siRNA Chimera Inhibitors of Intimal Hyperplasia 2012
  • Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to Adeno-Associated Virus Vector Containing a CMV Enhancer/Chicken beta-Actin Cassette in Murine Pompe Disease 2012
  • Increased Frequency of Permanent Integration Following Liver-Directed Gene Therapy with rDNA-AAV2/8 In Murine Phenylketonuria 2012
  • Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating 2012
  • Desensitization through Immunomodulatory Gene Therapy Suppresses the Antibody Response to Enzyme Replacement Therapy in Murine Pompe Disease 2009
  • Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells 2009
  • Immune Reconstitution after Gene Therapy for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID) 2009
  • Inflammatory Killing of the Pancreas Induces Multiple Immune Checkpoints Which Protect from Autoimmunity but Which Can Be Manipulated To Achieve Anti Tumor Immunity 2009
  • Mechanism of Shortened Bones in Mucopolysaccharidosis VII 2009
  • Modulation of CD4(+)CD25(+)Foxp3(+) Regulatory T Cells in the Recipients Is Necessary for Enhancing Anti-Tumor Immunity Following Stem Cell Transplantation 2009
  • RNA Transfected Dendritic Cell Vaccines Targeting Human Cytomegalovirus Antigens in Patients with Glioblastoma 2009
  • The TLR9-MyD88 Pathway Is Critical for Adaptive Immune Responses to AAV Vectors in Gene Therapy 2009
  • 1028. Use of Antisense Oligonucleotides To Restore Activity in Mutant p53 2006
  • 104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle 2006
  • 1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological Reconstitution 2006
  • 19. A New, Plasmid-Based Adenoviral Vectoring System Derived from the Highly Immunogenic, Human Ad4 Strain 2006
  • 211. Development of Helper-Dependent Adenovirus (HDAd) Vectors for Gene Therapy in the Mouse Model for Glycogen Storage Disease Type Ia (GSD-Ia) 2006
  • 487. Liver-Targeted Gene Therapy in Glycogen Storage Disease Type Ia (GSD-Ia) Requires Widespread, Regulated Glucose-6-phosphatase 2006
  • 563. Correction of Glycogen Storage Disease Type II by Systemic Delivery of an AAV2/8 Vector Containing a Muscle-Specific Promoter 2006
  • 586. Anti-HIV-1 Neutralizing Antibody Gene Delivery to Rhesus Macaques and Rodents: A Novel Vaccination Approach 2006
  • 7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry 2006
  • 739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue 2006
  • 83. Cell_Type Specific Delivery of siRNAs with Aptamer_siRNA Chimeras 2006
  • From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop. 2005
  • 339. Sustained, High-Level Expression of Human Acid Alpha-Glucosidase and Correction of Glycogen Storage Disease Type II (GSD II) with an Adeno-Associated Virus 8 (AAV2/8) Vector Containing a Liver-Specific Promoter 2004
  • A central role of RNA in developing therapeutic vaccines for cancer and infectious diseases 2004
  • Correction of glycogen storage disease type II (GSD II) with an adeno-associated virus 8 (AAV2/8) vector 2004
  • Correction of glycogen storage disease type II by intramuscular administration of an adeno-associated virus 6 (AAV2/6) vector containing a muscle-specific promoter 2004
  • Development of adeno-associated virus (AAV) vectors for gene therapy in the mouse model for glycogen storage disease type Ia (GSD-Ia) 2004
  • Group I intron-mediated trans-splicing revision of mutant p53 RNA transcripts 2004
  • Insertional analyses in rhesus monkey blood cells after non-myeloablative hematopoietic stem cell marking with a therapeutic onco-retroviral vector for X-linked chronic granulomatous disease 2004
  • A clinical attempt to treat JAK3-deficient SCID using retroviral-mediated gene transfer to bone marrow CD34+ cells 2003
  • A genetically engineered plasmid encoding a zinc finger VEGF-activating transcription factor induces angiogenesis in the rabbits with hind-limb ischemia 2003
  • Correction of glycogen storage disease type II (GSD II) by intramuscular administration of an adeno-associated virus (AAV) vector pseudotyped as AAV6 2003
  • Corrective gene transfer into bone marrow CD34+cells for adenosine deaminase (ADA) deficiency: Results in four patients after one year of follow-up 2003
  • Discontinuation of PEG-ADA replacement therapy in a patient with ADA-deficiency previously treated with retroviral-mediated, T cell-directed gene therapy 2003
  • Serial real-time luminescence imaging profiles differential kinetics and biodistibution of transgene expression from AAV serotype 1-5 vectors following adult and neonatal gene transfer 2003
  • Transmembrane transport of marker molecules and cell membrane kinetics during electropermeabilization 2003