Gene Therapy

• 

  Publication Venue For

  • Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice..  25:297-311. 2018
  • Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure..  23:313-319. 2016
  • Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia..  19:443-452. 2012
  • Peptide affinity reagents for AAV capsid recognition and purification..  18:1020-1024. 2011
  • Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease..  17:1500-1505. 2010
  • Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino..  17:132-140. 2010
  • A small regulatory element from chromosome 19 enhances liver-specific gene expression..  16:43-51. 2009
  • Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition..  15:1411-1423. 2008
  • Wild-type adenoviruses from groups A-F evoke unique innate immune responses, of which HAd3 and SAd23 are partially complement dependent..  15:885-901. 2008
  • Rho-kinase as a novel gene therapeutic target in treatment of cold ischemia/reperfusion-induced acute lethal liver injury: effect on hepatocellular NADPH oxidase system.  14:1425-1433. 2007
  • Synergy of adoptive T-cell therapy and intratumoral suicide gene therapy is mediated by host NK cells..  14:998-1009. 2007
  • Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774).  14:281. 2007
  • Gene therapy progress and prospects: RNA aptamers..  14:283-291. 2007
  • Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774)).  13:1430. 2006
  • VSV-G pseudotyped, MuLV-based, semi-replication-competent retrovirus for cancer treatment..  13:1457-1470. 2006
  • Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia..  13:1281-1289. 2006
  • The perforin-dependent immunological synapse allows T-cell activation-dependent tumor targeting by MLV vector particles..  13:1166-1177. 2006
  • Inducible regulation of Runx2-stimulated osteogenesis..  13:873-882. 2006
  • Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria..  13:457-462. 2006
  • Induction of stable RNA interference in mammalian cells..  13:503-508. 2006
  • Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model..  11:1590-1598. 2004
  • Retroviral delivery of GAD-IgG fusion construct induces tolerance and modulates diabetes: a role for CD4+ regulatory T cells and TGF-β?.  11:1487-1496. 2004
  • Characterization of the distinct orthotopic bone-forming activity of 14 BMPs using recombinant adenovirus-mediated gene delivery..  11:1312-1320. 2004
  • Induction of cell stress through gene transfer of an engineered heat shock transcription factor enhances tumor immunogenicity..  11:1099-1104. 2004
  • Pre-emptive gene therapy using recombinant adeno-associated virus delivery of extracellular superoxide dismutase protects heart against ischemic reperfusion injury, improves ventricular function and prolongs survival..  11:962-969. 2004
  • Focused ultrasound (HIFU) induces localized enhancement of reporter gene expression in rabbit carotid artery..  10:1600-1607. 2003
  • HSV-1 amplicon vector-mediated expression of ATM cDNA and correction of the ataxia-telangiectasia cellular phenotype..  10:1321-1327. 2003
  • Repeated intrathecal administration of plasmid DNA complexed with polyethylene glycol-grafted polyethylenimine led to prolonged transgene expression in the spinal cord..  10:1179-1188. 2003
  • Electromobility of plasmid DNA in tumor tissues during electric field-mediated gene delivery..  9:1286-1290. 2002
  • Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors..  9:1015-1022. 2002
  • Intra-arterial administration of a replication-selective adenovirus (dl1520) in patients with colorectal carcinoma metastatic to the liver: a phase I trial..  8:1618-1626. 2001
  • Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing..  7:1885-1895. 2000
  • Transcription factor decoy for NFkappaB inhibits TNF-alpha-induced cytokine and adhesion molecule expression in vivo..  7:1326-1332. 2000
  • An in vitro system for efficiently evaluating gene therapy approaches to hemoglobinopathies..  7:215-223. 2000
  • Adenovirus-mediated gene transfer of the beta2-adrenergic receptor to donor hearts enhances cardiac function..  6:1298-1304. 1999
  • Adenoviral reporter gene transfer to the human trabecular meshwork does not alter aqueous humor outflow. Relevance for potential gene therapy of glaucoma..  6:515-524. 1999
  • RNA repair as a novel approach to genetic therapy..  6:461-462. 1999
  • Tumor cells cotransduced with B7.1 and gamma-IFN induce effective rejection of established parental tumor..  6:253-262. 1999
  • Immunotherapy of cancer with dendritic cell-based vaccines..  5:1445-1446. 1998
  • Efficacy of multiple administrations of a recombinant adenovirus expressing wild-type p53 in an immune-competent mouse tumor model..  5:605-613. 1998
  • HVJ (Sendai virus)-cationic liposomes: a novel and potentially effective liposome-mediated technique for gene transfer to the airway epithelium.  4:631-638. 1997
  • Naked DNA delivered intraportally expresses efficiently in hepatocytes..  3:593-598. 1996
  • Transient immune blockade prevents formation of neutralizing antibody to recombinant adenovirus and allows repeated gene transfer to mouse liver..  3:412-420. 1996
  • Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo..  3:137-144. 1996