Gene Therapy

Palermo A, Doyonnas R, Bhutani N, Pomerantz J, Alkan O, Blau HM. 2009. Nuclear reprogramming in heterokaryons is rapid, extensive, and bidirectional. FASEB J 2009 Jan 13 [Epub ahead of print].

Pechan P, Rubin H, Lukason M, Ardinger J, DuFresne E, Hauswirth WW, Wadsworth SC, Scaria A. 2009. Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization. Gene Ther 16(1):10-6.

Chu Q, Moreland R, Yew NS, Foley J, Ziegler R, Scheule RK. 2008. Systemic insulin-like growth factor-1 reverses hypoalgesia and improves mobility in a mouse model of diabetic peripheral neuropath

Griesenbach U, Meng C, Farley R, Cheng SH, Scheule RK, Davies MH, Wolstenholme-Hogg PC, Ten Hove W, van der Hoeven P, Sinn PL, McCray PBJ, Inoue M, Geddes DM, Hasegawa M, Frankel G, Wiles S, Alton EW. 2008. In vivo imaging of gene transfer to the respiratory tract. Biomaterials 29:1533-1540.

Hyde SC, Pringle IA, Abdullah S, Lawton AE, Davies LA, Varathalingam A, Nunez-Alonso G, Green A-M, Bazzani RP, Sumner-Jones SG, Chan M, Li H, Yew NS, Cheng SH, Christopher Boyd A, Davies JC, Griesenbach U, Porteous DJ, Sheppard DN, Munkonge FM, Alton EWFW, Gill DR. 2008. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. Nature Biotech 26(5):549-51.

Jacobs F, Snoeys J, Feng Y, Van Craeyveld E, Lievens J, Armentano D, Cheng SH, De Geest B. 2008. Direct comparison of hepatocyte-specific expression cassettes following adenoviral and nonviral hydrodynamic gene transfer. Gene Ther 15(8):594-603.

Larsen MDB, Griesenbach U, Goussard S, Gruenert DC, Geddes DM, Scheule RK, Cheng SH, Courvalin P, Grillot-Courvalin C, Alton EWFW. 2008. Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli Gene Ther 15(6):434-42.

Natoli TA, Gareski TC, Dackowski WR, Smith L, Bukanov N, Russo RJ, Husson H, Matthews D, Piepenhagen PA, Ibraghimov-Beskrovnaya O. 2008. Pkd1 and Nek8 mutations affect cell-cell adhesion and cilia in cysts formed in kidney organ cultures. Am J Physiol Renal Physiol 294:F73-83.

O'Riordan CR, Song A. 2008. PEGylated adenovirus for targeted gene therapy. Methods Mol Biol 434:133-60.

Siders W, Shields J, Kaplan J, Lukason M, Woodworth L, Wadsworth S, Scaria A. 2008. Cytotoxic T-Lymphocyte (CTL) responses to the transgene product and not AAV capsid protein limit transgene expression in mice. Hum Gene Ther 2008 Oct 1. [Epub ahead of print].

Ziegler RJ, Bercury SD, Fidler J, Zhao MA, Foley J, Taksir TV, Ryan S, Hodges BL, Scheule RK, Shihabuddin LS, Cheng SH. 2008. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid α-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic pompe mice. Human Gene Therapy 19(6):609-21.

Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Ylä-Herttuala S. 2007. Progress and prospects: gene therapy clinical trials (part 1). Gene Ther 14:1439-1447.

Belanger AJ, Luo Z, Vincent KA, Akita GY, Cheng SH, Gregory RJ, Jiang C. 2007. Hypoxia-inducible factor 1 mediates hypoxia-induced cardiomyocyte lipid accumulation by reducing the DNA binding activity of peroxisome proliferator-activated receptor alpha/retinoid X receptor. Biochem Biophys Res Commun 364:567-572.

Davies LA, Seguela C, Varathalingam A, Cheng SH, Hyde SC, Gill DR. 2007. Identification of transfected cell types following non-viral gene transfer to the murine lung. J Gene Med 9:184-196.

De Bie C. 2007. Genzyme: 15 years of cell and gene therapy research. Regen Med 2:95-97.

Hughes-Wilson W, Mackay D. 2007. European approval system for advanced therapies: good news for patients and innovators alike. Regenerative Medicine. 2(1). . 5-6. 2:5-6.

McLachlan G, Baker A, Tennant P, Gordon C, Vrettou C, Renwick L, Blundell R, Cheng SH, Scheule RK, Davies L, Painter H, Coles RL, Lawton AE, Marriott C, Gill DR, Hyde SC, Griesenbach U, Alton EW, Boyd AC, Porteous DJ, Collie DD. 2007. Optimizing aerosol gene delivery and expression in the ovine lung. Mol Ther 15:348-354.

Parsons GB, Souza DW, Wu H, Yu D, Wadsworth S, Gregory R, Armentano D. 2007. Ectopic expression of glucagon-like peptide 1 for gene therapy of type II diabetes. Gene Ther 14:38-48.

Pringle IA, McLachlan G, Collie DD, Sumner-Jones SG, Lawton AE, Tennant P, Baker A, Gordon C, Blundell R, Varathalingam A, Davies LA, Schmid RA, Cheng SH, Porteous DJ, Gill DR, Hyde SC. 2007. Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung. J Gene Med 9:369-380.

Reczek D, Schwake M, Schröder J, Hughes H, Blanz J, Jin X, Brondyk W, Van Patten S, Edmunds T, Saftig P. 2007. LIMP-2 is a receptor for lysosomal mannose-6-phosphate-independent targeting of beta-glucocerebrosidase. Cell 131:770-783.

Vincent KA, Jiang C, Boltje I, Kelly RA. 2007. Gene therapy progress and prospects: Therapeutic angiogenesis for ischemic cardiovascular disease. Gene Ther 14:781-789.

Xenariou S, Griesenbach U, Liang HD, Zhu J, Farley R, Somerton L, Singh C, Jeffery PK, Ferrari S, Scheule RK, Cheng SH, Geddes DM, Blomley M, Alton EW. 2007. Use of ultrasound to enhance nonviral lung gene transfer in vivo. Gene Ther 14:768-774.

Yasuda M, Domaradzki ME, Armentano D, Cheng SH, Bishop DF, Desnick RJ. 2007. Acute intermittent porphyria: vector optimization for gene therapy. J Gene Med.

Ziegler RJ, Cherry M, Barbon CM, Li C, Bercury SD, Armentano D, Desnick RJ, Cheng SH. 2007. Correction of the biochemical and functional deficits in Fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A. Mol Ther 15:492-500.

Cachon-Gonzalez MB, Wang SZ, Lynch A, Ziegler R, Cheng SH, Cox TM. 2006. Effective gene therapy in an authentic model of Tay-Sachs-related diseases. Proc Natl Acad Sci U S A 103:10373-10378.

Griesenbach U, Kitson C, Escudero Garcia S, Farley R, Singh C, Somerton L, Painter H, Smith RL, Gill DR, Hyde SC, Chow YH, Hu J, Gray M, Edbrooke M, Ogilvie V, MacGregor G, Scheule RK, Cheng SH, Caplen NJ, Alton EW. 2006. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo. Respir Res 7:26.

Hodges BL, Cheng SH. 2006. Cell and gene-based therapies for the lysosomal storage diseases. Curr Gene Ther 6:227-241.

Kudo M, Brem MS, Canfield WM. 2006. Mucolipidosis II (I-cell disease) and Mucolipidosis IIIA (Classical Pseudo-Hurler Polydystrophy) are caused by mutations in the GlcNAc-phosphotransferase alpha / beta -subunits precursor gene. Am J Hum Genet 78:451-463.

Kudo M, Canfield WM. 2006. Structural requirements for efficient processing and activation of recombinant human UDP-N-acetylglucosamine: Lysosomal enzyme N-acetylglucosamine-1-phosphotransferase. J Biol Chem.

Luo Y, Jiang C, Belanger A, Akita G, Wadsworth S, Gregory R, Vincent K. 2006. A constitutively active HIF-1{alpha}/VP16 hybrid factor activates expression of the human B-Type natriuretic peptide gene. Mol Pharmacol.

McEachern KA, Nietupski JB, Chuang WL, Armentano D, Johnson J, Hutto E, Grabowski GA, Cheng SH, Marshall J. 2006. AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease. J Gene Med.

Passini MA, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG. 2006. Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. J Neurosci 26:1334-1342.

Plog MS, Guyre CA, Roberts BL, Goldberg M, George JA, Perricone MA. 2006. Preclinical safety and biodistribution of adenovirus-based cancer vaccines after intradermal delivery. Hum Gene Ther.

Xenariou S, Griesenbach U, Ferrari S, Dean P, Scheule RK, Cheng SH, Geddes DM, Plank C, Alton EW. 2006. Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo. Gene Ther.

Date T, Mochizuki S, Belanger AJ, Yamakawa M, Luo Z, Vincent KA, Cheng SH, Gregory RJ, Jiang C. 2005. Expression of constitutively stable hybrid hypoxia-inducible factor-1alpha protects cultured rat cardiomyocytes against simulated ischemia-reperfusion injury. Am J Physiol Cell Physiol 288:C314-320.

Dodge JC, Clarke J, Song A, Bu J, Yang W, Taksir TV, Griffiths D, Zhao MA, Schuchman EH, Cheng SH, O'Riordan CR, Shihabuddin LS, Passini MA, Stewart GR. 2005. Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease. Proc Natl Acad Sci U S A. 102:17822-17827.

Kaplan JM. 2005. Adenovirus-based cancer gene therapy. Curr Gene Ther 5:595-605.

Chu Q, Joseph M, Przybylska M, Yew NS, Scheule RK. 2005. Transient siRNA-mediated attenuation of liver expression from an alpha-galactosidase a plasmid reduces subsequent humoral immune responses to the transgene product in mice. Mol Ther 12:264-273.

Heinl-Green A, Radke PW, Munkonge FM, Frass O, Zhu J, Vincent K, Geddes DM, Alton EW. 2005. The efficacy of a 'master switch gene' HIF-1alpha in a porcine model of chronic myocardial ischaemia. Eur Heart J 26:1327-1332.

Hodges BL, Taylor KM, Chu Q, Scull SE, Serriello RG, Anderson SC, Wang F, Scheule RK. 2005. Local delivery of a viral vector mitigates neutralization by antiviral antibodies and results in efficient transduction of rabbit liver. Mol Ther 12:1043-1051.

Lynch CM, Johnson J, Vaccaro C, Thurberg BL. 2005. High-resolution light microscopy (HRLM) and digital analysis of Pompe disease pathology. J Histochem & cytochem 53:63-73.

Schreiber RE, Blease K, Ambrosio A, Amburn E, Sosnowski B, Sampath TK. 2005. Bone induction by AdBMP-2/collagen implants. J Bone Joint Surg Am 87:1059-1068.

Yew NS. 2005. Controlling the kinetics of transgene expression by plasmid design. Adv Drug Deliv Rev 57:769-780.

Yew NS, Cheng SH. 2005. Reducing the immunostimulatory activity of CpG-containing plasmid DNA vectors for non-viral gene therapy. Exp Opin Drug Deliv 2:199 (erratum).

Yew NS, Scheule RK. 2005. Toxicity of cationic lipid-DNA complexes. Adv Genet 53:189-214.

Braudeau C, Bouchet D, Tesson L, Iyer S, Remy S, Buelow R, Anegon I, Chauveau C. 2004. Induction of long-term cardiac allograft survival by heme oxygenase-1 gene transfer. Gene Ther 11:701-710.

Hodges BL, Taylor KM, Joseph MF, Bourgeois SA, Scheule RK. 2004. Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides. Mol Ther 10:269-278.

Jiang C, Yang Y-F, Cheng SH. 2004. Fas ligand gene therapy for vascular intimal hyperplasia. Curr Gene Ther 4:33-39.

Luo Z, Palasis M, Yamakawa M, Liu LX, Vincent KA, Trudell L, Akita GA, Koch WJ, Cheng SH, Gregory RJ, Jiang C. 2004. Catheter-mediated delivery of adenoviral vectors expressing beta-adrenergic receptor kinase C-terminus inhibits intimal hyperplasia and luminal stenosis in rabbit iliac arteries. J Gene Med 6:1061-1068.

Marshall J, Yew NS, Cheng SH. 2004. Formulation of synthetic gene delivery vectors for transduction of the airway epithelium. Methods Mol Biol 245:95-114.

Pechan P, Lukason MJ, Rubin HL, Scaria A, Wadsworth SC. 2004. Intraocular gene delivery of soluble VEGF receptors. Iovs 45.

Yew NS, Cheng SH. 2004. Reducing the immunostimulatory activity of CpG-containing plasmid DNA vectors for non-viral gene therapy. Exp Opin on Drug Del 1:115-125.

Yew NS, Cheng SH. 2004. Prospects for clinical application of synthetic gene delivery vectors. In: Gene & Cell Ther (2nd Ed). p 761-772.

Zhao H, Hemmi H, Akira S, Cheng SH, Scheule RK, Yew NS. 2004. Contribution of Toll-like receptor 9 signaling to the acute inflammatory response to nonviral vectors. Mol Ther 9:241-248.

Cheng SH, Smith AE. 2003. Gene therapy progress and prospects: gene therapy of lysosomal storage disorders. Gene Ther 10:1275-1281.

Date T, Luo Z, Yamakawa M, Belanger AJ, Scaria A, Cheng SH, Gregory RJ, Mochizuki S, Jiang C. 2003. Myocardial expression of baculoviral p35 alleviates doxorubicin-induced cardiomyopathy in rats. Hum Gene Ther 14:947-957.

Emerson M, Renwick L, Tate S, Rhind S, Milne E, Painter HA, Boyd AC, McLachlan G, Griesenbach U, Cheng SH, Gill DR, Hyde SC, Baker A, Alton EW, Porteous DJ, Collie DD. 2003. Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segments. Mol Ther 8:646-653.

Hodges BL, Scheule RK. 2003. Hydrodynamic delivery of DNA. Expert Opin Biol Ther 3:911-918.

Lanciotti J, Song A, Doukas J, Sosnowski B, Pierce G, Gregory R, Wadsworth S, O'Riordan C. 2003. Targeting adenoviral vectors using heterofunctional polyethylene glycol FGF2 conjugates. Mole Ther 8:99-107.

O'Riordan CR, Song A, Lanciotti J. 2003. Strategies to adapt adenoviral vectors for targeted delivery. Methods Mol Med 76:89-112.

St George JA. 2003. Gene therapy progress and prospects: adenoviral vectors. Gene Ther 10:1135-1141.

Tollefson AE, Scaria A, Ying B, Wold WS. 2003. Mutations within the ADP (E3-11.6K) protein alter processing and localization of ADP and the kinetics of cell lysis of adenovirus-infected cells. J Virol 77:7764-7778.

Tousignant JD, Zhao H, Yew NS, Cheng SH, Eastman SJ, Scheule RK. 2003. DNA sequences in cationic lipid:pDNA-mediated systemic toxicities. Hum Gene Ther 14:203-214.

Yamakawa M, Liu LX, Date T, Belanger AJ, Vincent KA, Akita GY, Kuriyama T, Cheng SH, Gregory RJ, Jiang C. 2003. Hypoxia-inducible factor-1 mediates activation of cultured vascular endothelial cells by inducing multiple angiogenic factors. Circ Res 93:664-673.

Yew NS, Cheng SH. 2003. Sustaining transgene expression in vivo. In: Rolland A, Sullivan SM, editors. Pharmaceutical Gene Delivery Systems: Marcel Dekker. p 17-46.

Date T, Belanger AJ, Mochizuki S, Sullivan JA, Liu LX, Scaria A, Cheng SH, Gregory RJ, Jiang C. 2002. Adenovirus-mediated expression of p35 prevents hypoxia/reoxygenation injury by reducing reactive oxygen species and caspase activity. Cardiovasc Res 55:309-319.

Eastman SJ, Baskin KM, Hodges BL, Chu Q, Gates A, Dreusicke R, Anderson S, Scheule RK. 2002. Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA. Hum Gene Ther 13:2065-2077.

Marshall J, Cheng SH. 2002. Formulation of synthetic vectors for cystic fibrosis gene therapy. Meth Mole Med 70:585-598.

O'Riordan C. 2002. Humoral immune response. In: Curiel DT, Douglas JT, editors. Adenoviral Vectors for Gene Therapy: Elsevier, Academic Press. p 375-407.

Ruzek MC, Kavanagh BF, Scaria A, Richards SM, Garman RD. 2002. Adenoviral vectors stimulate murine natural killer cell responses and demonstrate antitumor activities in the absence of transgene expression. Mole Ther 5:115-124.

Scheule RK. 2002. Cationic lipids for lung gene therapy. In: Albelda SM, editor. Lung Biology in Health and Disease: Marcel Dekker. p 93-118.

Tambuyzer E, Moll N, Muys P. 2002. EuropaBio's ethics committee and advisory group on ethics (AGE): an overview. J Biolaw Bus 5:56-58.

Yew NS, Zhao H, Przybylska M, Wu IH, Tousignant JD, Scheule RK, Cheng SH. 2002. CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol Ther 5:731-738.

Ziegler RJ, Li C, Cherry M, Zhu Y, Hempel D, van Rooijen N, Ioannou YA, Desnick RJ, Goldberg MA, Yew N, S., Cheng SH. 2002. Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease. Hum gene ther 13:935-945.