Research Topics
| Terence R FlotteSummaryAffiliation: University of Massachusetts Medical School Country: USA Publications
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Publications
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airwayTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
Mol Ther 18:594-600. 2010..T-cell responses to AAV5 capsid were stronger than AAV1 capsid. This dual vector indexing approach may be useful in selecting lead vector serotypes for clinical gene therapy and suggests rAAV1 is preferred for cystic fibrosis...
Preclinical study design for rAAVTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School Gene Therapy Center, Worcester, MA, USA
Methods Mol Biol 807:317-37. 2011....
In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negativeTerence R Flotte
University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
Mol Ther 16:806-7. 2008- Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim resultsTerence R Flotte
University of Massachusetts Medical School, 55 Lake Avenue North, Worcester, MA 01655, USA
Hum Gene Ther 22:1239-47. 2011..However, further improvements in the design or delivery of rAAV-AAT vectors will be required to achieve therapeutic target serum AAT concentrations... - Gene therapy for alpha-1 antitrypsin deficiencyTerence R Flotte
Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, S1 340, 55 Lake Avenue North, Worcester, MA 01655, USA
Hum Mol Genet 20:R87-92. 2011..Over the years, numerous strategies have been employed for the gene therapy of both AAT-deficient lung disease and liver disease. These will be reviewed with an emphasis on modalities that have reached clinical trials recently... - Gene therapy: the first two decades and the current state-of-the-artTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
J Cell Physiol 213:301-5. 2007.... - Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCADStuart G Beattie
University of Massachusetts Medical School, Worcester, MA 01655, USA
Hum Gene Ther 19:579-88. 2008..These results demonstrate biochemical correction of SCAD deficiency after AAV8-mediated SCAD gene delivery... - Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzeesTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA
Hum Gene Ther 19:681-9. 2008..These data indicate an increased susceptibility to subclinical liver toxicity from portal vein injection of rAAV2 in the presence of HCV infection... - Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sitesTerence R Flotte
Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL 32610, USA
Hum Gene Ther 18:245-56. 2007..The copy number of vector DNA in the blood and semen declined over time throughout the study. These two dose-dependent findings have served to guide to the design of a phase 1 human trial of rAAV1-AAT... - Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapyAllison M Keeler
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Mol Ther 20:1131-8. 2012..These promising results suggest rAAV9 gene therapy as a potential treatment for VLCAD deficiency in humans... - Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vectorThomas J Conlon
Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, and Department of Pediatrics, Children s Hospital of Pittsburgh, PA, USA
Hum Gene Ther 17:71-80. 2006..This study is the first to demonstrate the systemic correction of a fatty acid oxidation disorder with rAAV and the utility of MRS as a noninvasive method to monitor SCAD correction after in vivo gene therapy... - Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient miceStuart G Beattie
University of Massachusetts Medical School, Worcester, MA 01655, USA
J Gene Med 10:1113-23. 2008..Recombinant adeno-associated viral (rAAV) vectors with pseudotype capsids were investigated for their potential towards correcting the phenotype observed in mice heterozygous (+/-) for LCAD (i.e. liver and muscle steatosis)... - Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profilesChristian Mueller
Department of Pediatrics and Gene Therapy Center, UMass Medical School, Worcester, Massachusetts 01605, USA
Mol Ther 20:590-600. 2012..This safe dual-therapy approach can be applied to other disorders such as amyotrophic lateral sclerosis, Huntington disease, cerebral ataxia, and optic atrophies... - Long-term, efficient inhibition of microRNA function in mice using rAAV vectorsJun Xie
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Nat Methods 9:403-9. 2012..rAAV-mediated miRNA inhibition thus provides a simple way to study miRNA function in adult mammals and a potential therapy for dyslipidemia and other diseases caused by miRNA deregulation... - Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17eChristian Mueller
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA
Mol Ther 18:511-8. 2010..These studies demonstrate the feasibility of targeting T helper 2 (Th2) cytokines with rAAV-delivered fusion proteins as a means to treat aberrant immune responses... - Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapyTerence R Flotte
Department of Pediatrics, University of Florida, Gainesville, 32610, USA
Hum Gene Ther 16:921-8. 2005..The observation of sizeable physiological correction in the face of low mRNA levels may reflect the regulatory role of low levels of CFTR protein as an activator of other chloride channels... - Recombinant adeno-associated virus vectors for gene therapyThomas J Conlon
University of Florida College of Medicine, Department of Pediatrics, Box 100296, Gainesville, FL 32610-0296, USA
Expert Opin Biol Ther 4:1093-101. 2004..Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials... - Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neuronsKevin D Foust
Department of Pediatrics, Evelyn F and William L McKnight Brian Institute, University of Florida College of Medicine, Gainesville, FL 32610, USA
Hum Gene Ther 19:61-70. 2008..Our data suggest that systemic injection of rAAV8 is not an effective delivery route to target lower motor neurons, but could be useful for targeting sensory pathways in chronic pain... - Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responsesChristian Mueller
University of Massachusetts Medical School Department of Pediatrics and Gene Therapy Center, Worcester, Massachusetts 01605, USA
Am J Respir Cell Mol Biol 44:922-9. 2011..In summary, our data identified that CFTR dysfunction in T cells can lead directly to aberrant immune responses. These findings implicate the lymphocyte population as a potentially important target for CF therapeutics... - In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNAPedro E Cruz
Department of Pediatrics, University of Florida, Gainesville, FL, USA
Lab Invest 87:893-902. 2007..The rAAV8-3X-siRNA vector may hold promise as a potential therapy for patients with AAT liver disease... - CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetesMichael S Stalvey
Department of Pediatrics, University of Florida, College of Medicine, P O Box 100296, Gainesville, FL 32610, USA
Cytokine 44:154-9. 2008..These results suggest that, hyperglycemia may exacerbate the clinical course in CF by impacting immune reactivity. There is clear need to maximize metabolic management in CFRD... - Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the ratKevin D Foust
Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32611, USA
Hum Gene Ther 19:71-82. 2008..Spinal cord GDNF levels were elevated at distances up to 72 mm from the injection sites, and confirmed that RST-related GDNF transport to spinal cord surpassed CST-associated delivery... - Recent developments in recombinant AAV-mediated gene therapy for lung diseasesTerence R Flotte
Powell Gene Therapy Center, Genetics Institute, Department of Pediatrics, University of Florida, 1600 SW Archer Rd, Box 100296, Gainesville, FL 32610 0296, USA
Curr Gene Ther 5:361-6. 2005..In addition, the availability of an abundance of novel rAAV serotypes, each with its own receptor tropism, has expanded the range of possibilities for long-term success of gene therapy in the respiratory tract... - Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosisHong Yu
Department of Pediatrics, Medical University of South Carolina, 173 Ashley BSB 749, Charleston, SC 29403, USA
Am J Respir Cell Mol Biol 41:367-75. 2009..aeruginosa infection. This study demonstrates that the defective ASMase pathway in CF is a key contributor to the unabated IL-8 response with P. aeruginosa infection and to the compromised host response failing to eradicate bacteria... - Gene therapy for cystic fibrosisChristian Mueller
Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA, USA
Clin Rev Allergy Immunol 35:164-78. 2008..This learning curve has led to the optimization of vector technology and an appreciation of immune and mechanical barriers that have to be overcome for successful delivery... - Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adultsTerence R Flotte
Department of Pediatrics, University of Florida, Gainesville, USA
Hum Gene Ther 15:93-128. 2004.... - In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8Kirsten E Erger
Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA
J Gene Med 9:462-9. 2007..ATR functions within the mitochondria matrix in the final conversion of cobalamin into coenzyme B(12), adenosylcobalamin (AdoCbl). AdoCbl is a required coenzyme for the mitochondrial enzyme methylmalonyl-CoA mutase (MCM)... - Gene transfer in the lung using recombinant adeno-associated virusAlisha M Gruntman
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Curr Protoc Microbiol . 2012..Detailed procedures for lung delivery (intranasal, orotracheal, and surgical tracheal injection), sample collection, and post-mortem tissue processing will be described... - Production of clinical-grade recombinant adeno-associated virus vectorsRichard O Snyder
Powell Gene Therapy Center, University of Florida Genetics Institute, Department of Pediatrics, 1600 SW Archer Road, Gainesville, FL 32610-0266, USA
Curr Opin Biotechnol 13:418-23. 2002..The clinical manufacture of rAAV vectors has supported phase I and phase II trials, showing that adeno-associated virus serotype 2 vectors are safe when administered to humans... - Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectorsRenius Owen
Department of Pediatrics, the General Clinical Research Center, University of Florida, Gainesville, Florida 32610, USA
Mol Ther 6:394-9. 2002..These studies provide the basis for future efforts to develop a recombinant AAV (rAAV)-based gene therapy approach for the correction of PDC deficiency... - N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearanceAshley T Martino
Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida, USA
Am J Respir Cell Mol Biol 44:824-30. 2011..Augmenting N-glycosylation to attenuate colonization of P. aeruginosa in CF airways reveals a new therapeutic avenue for a hallmark disease condition in CF... - Viral vector-mediated and cell-based therapies for treatment of cystic fibrosisTerence R Flotte
Department of Pediatrics, University of Florida, Gainesville, Florida, USA
Mol Ther 15:229-41. 2007..This article will review the advancements made and challenges remaining in the development of viral vector-mediated and cell-based approaches to treat patients with CF... - Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- miceChristian Muller
Department of Pediatrics, Center for Immunology and Transplantation and Genetics Institute, University of Florida College of Medicine, Gainesville, FL 32610 0296, USA
Lab Invest 86:130-40. 2006..This system models a very specific type of airway inflammation in CF and could provide insights into pathogenesis and treatment of the disease... - Adeno-associated virus-based gene therapy for inherited disordersTerence R Flotte
Department of Pediatrics, University of Florida, Gainesville 32610, USA
Pediatr Res 58:1143-7. 2005..The evaluation of safety and efficacy of these newer agents is ongoing... - Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virusAlisha M Gruntman
Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts, USA
Curr Protoc Microbiol . 2013.... - The signal and the trap: targeted delivery and retention of proteins in the mitochondrionTerence R Flotte
Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville 32611, USA
Mol Ther 7:715-6. 2003 - MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expressionJun Xie
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
Mol Ther 19:526-35. 2011..Our findings promise to facilitate the development of miRNA-regulated rAAV for CNS-targeted gene delivery and other applications... - Endocrine parameters of cystic fibrosis: back to basicsMichael S Stalvey
Department of Pediatrics and the Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
J Cell Biochem 108:353-61. 2009..In this review, we focus on the known and hypothesized pathogenesis of these two disorders. Additionally, the molecular underpinnings of CF will be explained along with the potential interactions with endocrine disease phenotypes... - Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategiesJared N Silver
University of Florida College of Medicine, Department of Pediatrics, Gainesville, FL 32607, USA
Pharmacogenomics 9:947-68. 2008..This review endeavors to describe ADA-SCID, the traditional treatments, previous retroviral gene therapies, and primarily, alternative recombinant adeno-associated virus-based strategies to remedy this potentially fatal genetic disease... - Adeno-associated virus: a ubiquitous commensal of mammalsTerence R Flotte
Department of Pediatrics, and Genetics Institute, University of Florida, Gainesville, FL 32610, USA
Hum Gene Ther 16:401-7. 2005 - Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndromeAllison M Keeler
Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA
Hum Gene Ther 23:548-56. 2012..Important achievements have been made and have yet to come for cell and gene therapies for disorders of the lung and those mimicking SIDS... - Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell LineIsabel Virella-Lowell
Department of Pediatrics, Medical University of South Carolina, Charleston, SC 29401, USA
Mol Ther 10:562-73. 2004..In conclusion, the CFTR genotype changes the expression of multiple genes at baseline and in response to bacterial challenge, and only a subset of these changes is secondary to IL-10 deficiency... - Improved method of recombinant AAV2 delivery for systemic targeted gene therapyCathryn Mah
Department of Pediatrics, University of Florida, Gainesville, Florida 32610, USA
Mol Ther 6:106-12. 2002..These studies demonstrate a novel method to deliver rAAV vectors more effectively that could prove to be a successful alternative mode of virus-mediated human gene therapy... - Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiencyTerence R Flotte
Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610-0266, USA
Chest 121:98S-102S. 2002 - Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vectorThomas J Conlon
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32608, USA
Mol Ther 12:867-75. 2005..Therefore, pseudotyped AAV8 provides a vehicle to infect a high percentage of hepatocytes stably and thereby express therapeutic molecules to modify AAT PiZ transcripts... - Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical studyTerence R Flotte
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610 0296, USA
Hum Gene Ther 14:1079-88. 2003..These data indicate the need for continued evaluation of rAAV-CFTR vectors in additional clinical trials... - Systemic overexpression of IL-10 induces CD4+CD25+ cell populations in vivo and ameliorates type 1 diabetes in nonobese diabetic mice in a dose-dependent fashionKevin S Goudy
Department of Pathology, Powell Gene Therapy Center, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA
J Immunol 171:2270-8. 2003..This study indicates the potential for immunomodulatory gene therapy to prevent autoimmune diseases, including type 1 diabetes, and implicates IL-10 as a molecule capable of increasing the percentages of regulatory cells in vivo... - Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vectorYuanqing Lu
Department of Pharmaceutics, University of Florida College of Pharmacy, Gainesville, FL 32610, USA
J Gene Med 8:730-5. 2006..These results provide strong support for the functionality of AAT in ongoing clinical studies of muscle-directed AAT gene therapy... - Localized gene expression following administration of adeno-associated viral vectors via pancreatic ductsScott A Loiler
Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA
Mol Ther 12:519-27. 2005..This study demonstrates that rAAV vectors can be designed to deliver therapeutic genes efficiently to the pancreas and achieve high levels of gene expression and may be useful in treating pancreatic disorders, including T1D... - Cystic fibrosis transmembrane conductance regulator deficiency exacerbates islet cell dysfunction after beta-cell injuryMichael S Stalvey
Department of Pathology, University of Florida, College of Medicine, PO Box 100275, Gainesville, FL 32610, USA
Diabetes 55:1939-45. 2006..These studies suggest metabolic derangements in CFRD originate from an islet dysfunction inherent to the CFTR(-/-) state... - Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vectorJeffrey Sirninger
Powell Gene Therapy Center, UF Genetics Institute, Department of Pediatrics and Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL 32619-0296, USA
Hum Gene Ther 15:832-41. 2004..These studies provide functional characterization of a new version of rAAV-CFTR vectors... - A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomiesJohn A Wagner
Department of Molecular Pharmacology, Stanford CCSR Building, Stanford, CA 94305-5174, USA
Hum Gene Ther 13:1349-59. 2002..In summary, this Phase II trial confirms the safety of tgAAVCF but provides little support of its efficacy in the within-patient controlled sinus study. Various potentially confounding factors are discussed... - Adeno-associated viral vectors for CF gene therapyTerence R Flotte
Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA
Methods Mol Med 70:599-608. 2002 - Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspectiveTerence R Flotte
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610-0296, USA
Hum Gene Ther 15:716-7. 2004 - Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vectorMatthias Kapturczak
Department of Medicine, University of Florida, Gainesville, Florida 32610, USA
Mol Ther 5:154-60. 2002..These data are the first report of efficient islet cell transduction with two genes using a single bicistronic rAAV vector and have direct implications for strategies aimed at enhancing islet transplant survival... - The murine alpha(1)-proteinase inhibitor gene family: polymorphism, chromosomal location, and structureKaren W Barbour
Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, 29208, USA
Genomics 80:515-22. 2002..Finally, we present the complete sequence of an 84-kb region of Serpina1 containing a tandem repeat of two alpha(1)-PI genes... - Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaquesAnne C Fischer
Department of Physiology, The Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA
Mol Ther 8:918-26. 2003..This study is the first to demonstrate successful gene transfer subsequent to repeated aerosolized doses of rAAV2 in immunocompetent nonhuman primates without associated inflammatory responses prohibitive to transgene expression... - The pyruvate dehydrogenase complex as a target for gene therapyPeter W Stacpoole
Department of Medicine, Division of Endocrinology and Metabolism, University of Florida, College of Medicine, Gainesville, FL 32610, USA
Curr Gene Ther 3:239-45. 2003.... - Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectorsSifeng Chen
Department of Medicine, University of Florida, Gainesville, Florida 32610, USA
J Am Soc Nephrol 14:947-58. 2003.... - Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaquesSuzanne E Beck
Eudowood Division of Pediatric Respitarory Sciences and Departments of Pediatrics, and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA
Mol Ther 6:546-54. 2002..u. (P=0.030). We conclude that expression of rAAV2-GFP in lungs appears to be related to depositing a regional threshold dose greater than 3 x 10(9) i.u., easily achieved by bronchoscopic microspraying... - A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibitionByoung Boo Seo
Division of Biochemistry, Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, California 92037, USA
Mol Ther 6:336-41. 2002..It is conceivable that the NDI1 gene will be a promising tool in the treatment of neurodegenerative conditions caused by complex I inhibition... - Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspectsSihong Song
Powell Gene Therapy Center of the University of Florida Genetics Institute, University of Florida, Gainesville, Florida 32615, USA
Mol Ther 6:329-35. 2002..These studies indicate that the risks of immune reaction and germline transmission after intramuscular injection of rAAV-SERPINA1 in nonhuman primates are relatively low within the range of vector doses studied... - Virus-based gene delivery systemsCathryn Mah
Powell Gene Therapy Center, University of Florida, Gainesville, Florida 32610-0266, USA
Clin Pharmacokinet 41:901-11. 2002..This review will also illustrate several current improved physical delivery systems for optimal vector administration... - Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kineticsArtur V Cideciyan
Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Proc Natl Acad Sci U S A 105:15112-7. 2008..Cone-sensitivity recovery time was rapid. These results demonstrate dramatic, albeit imperfect, recovery of rod- and cone-photoreceptor-based vision after RPE65 gene therapy... - Functional expression of the single subunit NADH dehydrogenase in mitochondria in vivo: a potential therapy for complex I deficienciesByoung Boo Seo
Division of Biochemistry, Department of Molecular and Experimental Medicine, Scripps Research Institute, La Jolla, CA 92037, USA
Hum Gene Ther 15:887-95. 2004..The data indicate that the NDI1 gene will be a promising therapeutic tool in the treatment of encephalomyopathies and neurodegenerative diseases caused by complex I impairments... - Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosisSamuel G Jacobson
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Gene Ther 17:845-58. 2006..RPE65. The potential value of foveal treatment for LCA and other retinal degenerations warrants further research into how to achieve gene transfer without retinal injury from surgical detachment of the retina... - Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic painErin D Milligan
Department of Psychology and the Center for Neuroscience, University of CO at Boulder, Boulder, CO 80309, USA
Pain 126:294-308. 2006..Together, these data suggest that intrathecal IL-10 gene therapy may provide a novel approach for prolonged clinical pain control... - alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosisIrina Petrache
Department of Medicine, The Johns Hopkins University School of Medicine, Baltimore, MD, USA
Am J Pathol 169:1155-66. 2006.... - Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adultsMark L Brantly
Department of Medicine, University of Florida, Gainesville, FL 32611, USA
Hum Gene Ther 17:1177-86. 2006.... - Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionSamuel G Jacobson
Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Ther 13:1074-84. 2006..5-log unit range of vector doses proved efficacious. The efficacy and toxicity limits defined in this study lead to suggestions for the design of a subretinal AAV-2/2.RPE65 human trial of RPE65-associated LCA... - Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathwaySifeng Chen
Department of Medicine, Nephrology Research and Training Center, University of Alabama at Birmingham, Birmingham, AL 35294
Proc Natl Acad Sci U S A 102:7251-6. 2005.... - The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cellsAgnieszka Swiatecka-Urban
Department of Physiology, Dartmouth Medical School, Hanover, New Hampshire 03755, USA
J Biol Chem 280:36762-72. 2005.... - The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiencyPedro E Cruz
University of Florida, Nephrology Division, College of Medicine, FL, USA
Pharmacogenomics 8:1191-8. 2007..The last two trials delivered recombinant adeno-associated vectors encoding the alpha-1 antitrypsin cDNA by intramuscular injection. In this review, the progress of ongoing clinical trials and new gene therapy technologies is discussed... - Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR geneChristian Mueller
Department of Pediatrics and Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, FL, USA
J Gene Med 10:51-60. 2008..Gene therapy with rAAV5Delta-264CFTR attenuated the hyper-IgE response in this reproducible CF mouse model of ABPA, with systemic effects also evident in the cytokine response of stimulated splenocytes... - Adeno-associated virus-mediated gene transfer for lung diseasesTerence R Flotte
Powell Gene Therapy Center, Genetics Institute, and Department of Pediatrics, University of Florida, Gainesville, FL 32610, USA
Hum Gene Ther 16:643-8. 2005 - Enhancing rAAV vector expression in the lungIsabel Virella-Lowell
Department of Pediatrics of the University of Florida College of Medicine, USA
J Gene Med 7:842-50. 2005..Although the advantage of rAAV5 over rAAV2 in the lung has already been described, the availability of another serotype (rAAV1) capable of efficient gene transfer in the lung could be useful... - Controlling neuropathic pain by adeno-associated virus driven production of the anti-inflammatory cytokine, interleukin-10Erin D Milligan
Department of Psychology and the Center for Neuroscience, University of CO at Boulder, Boulder, CO 80309 USA
Mol Pain 1:9. 2005..Taken together, these data provide initial support that intrathecal gene therapy to drive the production of IL-10 may prove to be an efficacious treatment for neuropathic pain... - Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectorsSifeng Chen
Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA
Hum Gene Ther 16:235-47. 2005..These results suggest the unique potential of rAAV1 and rAAV5-based vectors for vascular-targeted gene-based therapeutic strategies... - Herpesvirus-based infectious titering of recombinant adeno-associated viral vectorsImran Mohiuddin
Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL 32610-0266, USA
Mol Ther 11:320-6. 2005..These methods generate reliable infectious titers for AAV vectors of different serotypes, thus enhancing product characterization and reducing risk in future clinical applications... - Ex vivo transduced liver progenitor cells as a platform for gene therapy in miceSihong Song
Department of Pharmaceutics, University of Florida, Gainesville, FL 32610, USA
Hepatology 40:918-24. 2004.... - IL-10 regulation of lupus in the NZM2410 murine modelKim R M Blenman
Department of Pathology, Immunology, and Laboratory Medicine, University of Florida, Gainesville, FL 32601-0275, USA
Lab Invest 86:1136-48. 2006..These results also reinforce the notion that IL-10 exerts multiple functions and commend caution in equating high levels of IL-10 and increased pathogenesis in systemic autoimmunity...