Publications


Publications Contributed To

  1. Wu P, Xiao W, Conlon T, et al. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropismJ Virol. 2000;74(18):8635–8647. doi:10.1128/jvi.74.18.8635-8647.2000
  2. Owen R 4th, Mandel RJ, Ammini CV, et al. Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectorsMol Ther. 2002;6(3):394–399. doi:10.1006/mthe.2002.0683
  3. Loiler SA, Conlon TJ, Song S, et al. Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liverGene Ther. 2003;10(18):1551–1558. doi:10.1038/sj.gt.3302046
  4. Conlon TJ, Flotte TR. Recombinant adeno-associated virus vectors for gene therapyExpert Opin Biol Ther. 2004;4(7):1093–1101. doi:10.1517/14712598.4.7.1093
  5. Virella-Lowell I, Zusman B, Foust K, et al. Enhancing rAAV vector expression in the lungJ Gene Med. 2005;7(7):842–850. doi:10.1002/jgm.759
  6. Conlon TJ, Cossette T, Erger K, et al. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vectorMol Ther. 2005;12(5):867–875. doi:10.1016/j.ymthe.2005.05.016
  7. Conlon TJ, Walter G, Owen R, et al. Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vectorHum Gene Ther. 2006;17(1):71–80. doi:10.1089/hum.2006.17.71
  8. Brantly ML, Spencer LT, Humphries M, et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adultsHum Gene Ther. 2006;17(12):1177–1186. doi:10.1089/hum.2006.17.1177
  9. Pacak CA, Mah CS, Thattaliyath BD, et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivoCirc Res. 2006;99(4):e3–e9. doi:10.1161/01.RES.0000237661.18885.f6
  10. Jacobson SG, Boye SL, Aleman TS, et al. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosisHum Gene Ther. 2006;17(8):845–858. doi:10.1089/hum.2006.17.845
  11. Jacobson SG, Acland GM, Aguirre GD, et al. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionMol Ther. 2006;13(6):1074–1084. doi:10.1016/j.ymthe.2006.03.005
  12. Flotte TR, Conlon TJ, Poirier A, Campbell-Thompson M, Byrne BJ. 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 sitesHum Gene Ther. 2007;18(3):245–256. doi:10.1089/hum.2006.113
  13. Andino LM, Conlon TJ, Porvasnik SL, Boye SL, Hauswirth WW, Lewin AS. Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virusGenet Vaccines Ther. 2007;5:13. Published 2007 Dec 10. doi:10.1186/1479-0556-5-13
  14. Erger KE, Conlon TJ, Leal NA, Zori R, Bobik TA, Flotte TR. In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8J Gene Med. 2007;9(6):462–469. doi:10.1002/jgm.1040
  15. Beattie SG, Goetzman E, Conlon T, et al. Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCADHum Gene Ther. 2008;19(6):579–588. doi:10.1089/hum.2007.168
  16. Flotte TR, Goetzmann J, Caridi J, et al. Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzeesHum Gene Ther. 2008;19(7):681–689. doi:10.1089/hum.2007.174
  17. Beattie SG, Goetzman E, Tang Q, et al. Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient miceJ Gene Med. 2008;10(10):1113–1123. doi:10.1002/jgm.1242
  18. Hauswirth WW, Aleman TS, Kaushal S, et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trialHum Gene Ther. 2008;19(10):979–990. doi:10.1089/hum.2008.107
  19. Pacak CA, Conlon T, Mah CS, Byrne BJ. Relative persistence of AAV serotype 1 vector genomes in dystrophic muscleGenet Vaccines Ther. 2008;6:14. Published 2008 Oct 15. doi:10.1186/1479-0556-6-14
  20. Cideciyan AV, Hauswirth WW, Aleman TS, et al. Vision 1 year after gene therapy for Leber’s congenital amaurosisN Engl J Med. 2009;361(7):725–727. doi:10.1056/NEJMc0903652
  21. Brantly ML, Chulay JD, Wang L, et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy [published correction appears in Proc Natl Acad Sci U S A. 2009 Oct 13;106(41):17606]. Proc Natl Acad Sci U S A. 2009;106(38):16363–16368. doi:10.1073/pnas.0904514106
  22. Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, et al. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteinsAnn Neurol. 2009;66(3):290–297. doi:10.1002/ana.21732
  23. Cideciyan AV, Hauswirth WW, Aleman TS, et al. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 yearHum Gene Ther. 2009;20(9):999–1004. doi:10.1089/hum.2009.086
  24. Mah CS, Falk DJ, Germain SA, et al. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established diseaseMol Ther. 2010;18(3):502–510. doi:10.1038/mt.2009.305
  25. Lock M, McGorray S, Auricchio A, et al. Characterization of a recombinant adeno-associated virus type 2 Reference Standard MaterialHum Gene Ther. 2010;21(10):1273–1285. doi:10.1089/hum.2009.223
  26. Kang HR, Gjorgjieva M, Smith SN, et al. Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage DiseaseMol Ther Methods Clin Dev. 2019;15:383–391. Published 2019 Nov 11. doi:10.1016/j.omtm.2019.10.016
  27. Mendell JR, Rodino-Klapac LR, Rosales XQ, et al. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2DAnn Neurol. 2010;68(5):629–638. doi:10.1002/ana.22251
  28. Silver JN, Elder M, Conlon T, et al. Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiencyHum Gene Ther. 2011;22(8):935–949. doi:10.1089/hum.2010.121
  29. Specht A, Fiske L, Erger K, et al. Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver diseaseJ Biomed Biotechnol. 2011;2011:646257. doi:10.1155/2011/646257
  30. Byrne BJ, Falk DJ, Pacak CA, et al. Pompe disease gene therapyHum Mol Genet. 2011;20(R1):R61–R68. doi:10.1093/hmg/ddr174
  31. Boye SL, Conlon T, Erger K, et al. Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouseInvest Ophthalmol Vis Sci. 2011;52(10):7098–7108. Published 2011 Sep 9. doi:10.1167/iovs.11-7867
  32. Jacobson SG, Cideciyan AV, Ratnakaram R, et al. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsArch Ophthalmol. 2012;130(1):9–24. doi:10.1001/archophthalmol.2011.298
  33. Polyak S, Mach A, Porvasnik S, et al. Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitisAm J Physiol Gastrointest Liver Physiol. 2012;302(3):G296–G308. doi:10.1152/ajpgi.00562.2010
  34. Keeler AM, Conlon T, Walter G, et al. Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy [published correction appears in Mol Ther. 2012 Jun;20(6):1287]. Mol Ther. 2012;20(6):1131–1138. doi:10.1038/mt.2012.39
  35. Lee NC, Falk DJ, Byrne BJ, et al. An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic deliveryGenet Vaccines Ther. 2012;10(1):3. Published 2012 Jun 18. doi:10.1186/1479-0556-10-3
  36. ElMallah MK, Falk DJ, Lane MA, et al. Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9Hum Gene Ther Methods. 2012;23(2):148–156. doi:10.1089/hgtb.2012.009
  37. Boye SE, Alexander JJ, Boye SL, et al. The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retinaHum Gene Ther. 2012;23(10):1101–1115. doi:10.1089/hum.2012.125

Abstracts

* Presented by grad student or medical student

  1. Sosa I, DVM, University of Florida; Winter MD, DVM, DACVR, University of Florida; Erger K, MBA, University of Florida; Mincey B, BS, University of Florida; Shih A, DVM; DACVA; Conlon TJ, PhD, University of Florida; Estrada AH, DVM, ACVIM (Cardiology), University of Florida. Biodistribution of retrograde coronary sinus delivery of mesenchymal stem cells: a pilot study. NAVRMA 2012.
  2. *Allison M. Keeler, Thomas Conlon, Kirsten Erger, Scott Shaffer, Terence R. Flotte. Sex-Dependent Expression of Very Long Chain acyl-CoA Dehydrogenase (VLCAD) Transgene and the Role of the Liver in biochemical and Phenotypic Correction of VLCAD Deficiency. ASGCT 2012.
  3. *Allison M. Keeler, Miguel Sena-Esteves, Thomas Conlon, Kirsten Erger, Fu Dongtao, Terence R. Flotte. Transduction and Expression in brain after Systemic Delivery of Single Stranded AAV9 in Adult Mice. ASGCT 2012.
  4. Shipla Prabhakar, Mehran Taherian, David Gianni, Thomas J. Conlon, Anat S. Rachamimov, Miguel S. Esteves, Xandra O. Breakefield, Gary J. Brenner. Regression of Schwannomas Induced by AAV-Mediated Delivery of Caspase-1. ASGCT 2012.
  5. *Darin J. Falk, Cathryn S. Mah, Meghan S. Soustek, Kun-Ze Lee, Denise A. Cloutier, Mai K. Elmallah, Kirsten E. Erger, Thomas J. Conlon, David D. Fuller, Barry J. Byrne. Intrathoracic Administration of AAV9 Reverses Neural and Cardiorespiratory Dysfunction in Pompe Disease. ASGCT 2011. Seattle, Washington.
  6. Sanford L. Boye, Thomas Conlon, Kirsten Erger, Travis Cossette, Jijing Pang, Sukanya Karan, Renee Ryals, Wolfgang Baehr, William W. Hauswirth, Shannon E. Boye. Long-Term Persistence of AAV Mediated Gene Therapy for Leber Congenital Amaurosis Type 1 (LCA1). ASGCT 2011.
  7. *Allison M. Keeler, Glenn Walters, Thomas Conlon, Karin Green, Christian Mueller, Terence R. Flotte. Long-Term Correction of Very Long-Chain Acyl-CoA Deydrogenase Deficiency by Recombinant Adeno-Associated Virus 9 Gene Therapy. ASGCT 2011. Seattle, Washington.
  8. Shannon E. Boye, Sanford L. Boye, Thomas Conlon, Kristin Erger, Jijing Pang, Xuan Liu, Sukanya Karan, Wolfgang Baehr, William W. Hauswirth. Gene Therapy for Gucy2d Leber congenital amaurosis (LCA1). ARVO 2011. Fort Lauderdale, Florida.
  9. A Specht, L Fiske, K Erger, T Cossette, A Thomas, J Verstegen, M Campbell-Thompson, MB Struck, BJ Byrne, CE Correia, CS Mah, DA Weinstein, TJ Conlon. Clinical Manifestations of Heterozygous Expression of a Mutation that Causes Glycogen Storage Disease Type IA in Dogs. ACVIM 2011. Denver, Colorado.
  10. Polyak S, Conlon T, Mach A, Porvasnik S, Wasserfall C, Dixon L, Mah C. Intestinal Directed Adeno-associated Virus Expressing IL-10 Improves Enterocolitis in a Murine Model of Inflammatory Bowel Disease. Digestive Disease Week AGA Meeting, New Orleans, LA 2010
  11. *Kirsten Erger, Stacy Porvasnik, Travis Cossette, Lynn Combee, Cheryl Roberts, Corinne Abernathy, Carol Detrisac, Thomas Conlon, Barry Byrne. Preclinical Toxicology and Biodistribution Study of Recombinant Adeno-associated Virus 1 (rAVV1) Human Acid a-Glucosidase (GAA) in NZW Rabbits by Intrathoracic Direct Injection in the Diaphragm. American Society of Gene Therapy 2009. San Diego, California.
  12. Mark L. Brantly, Margaret Humphries, Sihong Song, Thomas Conlon, Amy Poirier, Barry J. Byrne, Richard Snyder, Terence R. Flotte. Phase I Clinical Trial of Recombinant Adeno-Associated Virus (rAAV)-Alpha-1 Antitrypsin Vectors. Molecular Therapy 2005. American Society of Gene Therapy 2005. St. Louis, Missouri.
  13. Amy E. Poirier, Thomas J. Conlon, Lynn A. Combee, Kirsten E. Erger, Terence R. Flotte. Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 1 (rAAV1) Alpha-1 Antitrypsin (AAT) Vector. Molecular Therapy 2005. American Society of Gene Therapy 2005. St. Louis, Missouri.
  14. Kirsten E. Erger, Thomas J. Conlon, Travis L. Cossette, Thomas A. Bobik, Terence R. Flotte. In Vivo Expression of Human Cob(I)alamin Adenosyltransferase Using rAAV Serotypes 1,2 and 8, an Approach to Gene Therapy for Methylmalonic Aciduria (MMA). Molecular Therapy 2005. American Society of Gene Therapy 2005. St. Louis, Missouri.
  15. Brantly M, Humphries M, Song S, Conlon T, Poirier A, Byrne BJ, Snyder R and Flotte TR. Prelinical and Phase I/II Clinical Trials of Recombinant Adeno-Associated Virus (rAAV)-Alpha-1 Antitrypsin Vectors. 2005 Pediatric Academic Societies annual meeting. Washington, DC.

University of Florida Poster Presentations

  1. Wei F, Song S, Loiler S, Cruz P, Conlon T, Wei J, Zhao G, Flotte T. Recombinant Adeno-Associated Virus-Mediated Gene Transfer for Embryonic Stem Cell Gene Therapy. Pediatric Research Day 2003; 53:256A. Gainesville, Florida.
  2. Virella-Lowell I, Zuzman B, Conlon T, Morgan M, Song S, Flotte TR. A CMV/beta actin hybrid promoter improves rAAV vector expression in vitro and in vivo. Pediatric Research Day 2000; 47:483A. Gainesville, Florida.
  3. Conlon TJ, Brantly M, Lewin A, Flotte TR. A ribozyme-mediated gene therapy approach for neonatal liver disease due to PIZ mutant alpha 1-antitrypsin. Pediatric Research Day 2000; 47:239A. Gainesville, Florida.
  4. Thomas Conlon, Amara Estrada, Bijoy Thattaliyath, Stacy Porvasnik, Andre Shih, Travis Cossette, Sean Germain, Barry Byrne. Modified Skeletal Myoblast Therapy for Cardiac Failure Using AAV SDF1 in Rattus and Porcine Models. University of Florida Pediatrics Science Days. May 13, 2010.
  5. Thomas Conlon, Amara Estrada, Bijoy Thattaliyath, Stacy Porvasnik, Andre Shih, Travis Cossette, Sean Germain, Barry Byrne. Modified Skeletal Myoblast Therapy for Cardiac Failure Using AAV SDF1 in Rattus and Porcine Models. University of Florida College of Medicine Celebration of Research. April 19, 2010.
  6. Kirsten Erger, Stacy Porvasnik, Travis Cossette, Lynn Combee, Cheryl Roberts, Corinne Abernathy, Carol Detrisac, Thomas Conlon, Barry Byrne. Preclinical Toxicology and Biodistribution Study of Recombinant Adeno-associated Virus 1 (rAVV1) Human Acid a-Glucosidase (GAA) in NZW Rabbits by Intrathoracic Direct Injection in the Diaphragm. University of Florida Pediatrics Science Day, April 24th, 2009.
  7. Kirsten Erger, Lynn A Combee, Travis Cossette, Cheryl Roberts, Stacy Porvasnik, Denise Cloutier, Thomas J Conlon, Barry J Byrne. Toxicology, Biodistribution and Long Term Safety Study of a Recombinant AAV1 Vector for Limb Girdle Muscular Dystrophy (LGMD) TYPE-2D. University of Florida Pediatrics Science Day, March 13th, 2008.
  8. LM Fiske*, A Specht*, K Erger, T Cossette, A Thomas, J Verstegen, M Campbell-Thompson, MB Struck, BJ Byrne, CE Correia, CS Mah, TJ Conlon**, DA Weinstein** Evidence of Abnormal Growth and Metabolic Profiles in Heterozygotes in the Canine Model of Glycogen Storage Disease Type Ia. University of Florida Pediatrics Science Day, March 5th, 2012.

Poster Presentations

  1. Poirier A, Combee L, Martino A, Conlon T, and Flotte TR. Toxicology and biodistribution of AAV-AAT vectors. Presented at the American Society of Gene Therapy meeting, Minneapolis, MN, June 2-6, 2004.
  2. Poirier A, Combee L and Flotte TR. Toxicology and biodistribution of AAV-AAT vectors. Presented at the American College of Toxicology meeting, November 2-5, 2004, Washington D.C.
  3. Poirier AE, Conlon TJ, Combee LA, Erger KE, Flotte TR. Presented at the American Society of Gene Therapy conference, St. Louis, MO, June 1-5, 2005.

University of Florida Lecture

  1. Proof of Concept, Preclinical and Phase I Clinical Trials of rAAV-AAT. University of Florida Department of Pediatrics Bench to Bedside and Beyond. Jun 3, 2009.