Search Results - "Scheule, R.K."

Inhibition of Glycogen Biosynthesis via mTORC1 Suppression as an Adjunct Therapy for Pompe Disease by Ashe, K, Taylor, K.M, Chu, Q, Meyers, E, Ellis, A, Jingozyan, V, Klinger, K, Finn, P.F, Cooper, C.G.F, Chuang, W, Marshall, J, McVie-Wylie, A.J, McPherson, J.M, Mattaliano, R.J, Cheng, S.H, Scheule, R.K, Moreland, R.J

“…Methods, Results We have examined the biochemical pathways regulating glycogen accumulation in muscle tissue of GAA-/- mice, and evaluated the efficacy of…”
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Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung by McLachlan, G, Davidson, H, Holder, E, Davies, L A, Pringle, I A, Sumner-Jones, S G, Baker, A, Tennant, P, Gordon, C, Vrettou, C, Blundell, R, Hyndman, L, Stevenson, B, Wilson, A, Doherty, A, Shaw, D J, Coles, R L, Painter, H, Cheng, S H, Scheule, R K, Davies, J C, Innes, J A, Hyde, S C, Griesenbach, U, Alton, E W F W, Boyd, A C, Porteous, D J, Gill, D R, Collie, D D S

“…We use both large and small animal models in our pre-clinical evaluation of gene transfer agents (GTAs) for cystic fibrosis (CF) gene therapy. Here, we report…”
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Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung by Alton, E W F W, Boyd, A C, Cheng, S H, Davies, J C, Davies, L A, Dayan, A, Gill, D R, Griesenbach, U, Higgins, T, Hyde, S C, Innes, J A, McLachlan, G, Porteous, D, Pringle, I, Scheule, R K, Sumner-Jones, S

“…For gene therapy to improve lung function in cystic fibrosis (CF) subjects, repeated administration of the gene transfer agent over the lifetime of patients is…”
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Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease by Cabrera-Salazar, M.A., Bercury, S.D., Ziegler, R.J., Marshall, J., Hodges, B.L., Chuang, W.-L., Pacheco, J., Li, L., Cheng, S.H., Scheule, R.K.

“…Gaucher disease is caused by a deficit in the enzyme glucocerebrosidase. As a consequence, degradation of the glycolipids glucosylceramide (GluCer) and…”
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Use of ultrasound to enhance nonviral lung gene transfer in vivo by Xenariou, S, Griesenbach, U, Liang, H-D, Zhu, J, Farley, R, Somerton, L, Singh, C, Jeffery, P K, Ferrari, S, Scheule, R K, Cheng, S H, Geddes, D M, Blomley, M, Alton, E W F W

“…We have assessed if high-frequency ultrasound (US) can enhance nonviral gene transfer to the mouse lung. Cationic lipid GL67/pDNA, polyethylenimine (PEI)/pDNA…”
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Biophysical characterization of cationic lipid:DNA complexes by Eastman, S.J, Siegel, C, Tousignant, J, Smith, A.E, Cheng, S.H, Scheule, R.K

“…To better understand the structures formed by the interaction of cationic lipids with DNA, we undertook a systematic analysis to determine the biophysical…”
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Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli by Larsen, M D B, Griesenbach, U, Goussard, S, Gruenert, D C, Geddes, D M, Scheule, R K, Cheng, S H, Courvalin, P, Grillot-Courvalin, C, Alton, E W F W

“…Bacteria-mediated gene transfer (‘bactofection’) has emerged as an alternative approach for genetic vaccination and gene therapy. Here, we assessed…”
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EGTA enhancement of adenovirus-mediated gene transfer to mouse tracheal epithelium in vivo by Chu, Q, St George, J A, Lukason, M, Cheng, S H, Scheule, R K, Eastman, S J

“…Administration of recombinant adenoviral (AdV) vectors to animals can lead to inflammatory and immune responses. For therapeutic indications in which repeated…”
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33. Systemic Delivery of AAV8/DC190-GAA to Pompe Mice Normalizes Muscle Pathology and Corrects Motor Function Deficits by Ziegler, R.J., Zhao, M.A., Bercury, S.D., Fidler, J.A., Foley, J.W., Armentano, D., Ryan, S., Taksir, T., Griffiths, D., Shihabuddin, L.S., Scheule, R.K., Cheng, S.H.

“…Pompe disease, a lysosomal storage disorder, is generally known for it's characteristic degenerative myopathy. Massive accumulation of glycogen in lysosomes of…”
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Binding and uptake of cationic lipid:pDNA complexes by polarized airway epithelial cells by Chu, Q, Tousignant, J D, Fang, S, Jiang, C, Chen, L H, Cheng, S H, Scheule, R K, Eastman, S J

“…To better understand the barriers associated with cationic lipid-mediated gene transfer to polarized epithelial cells, Fischer rat thyroid (FRT) cells and…”
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Stoichiometry of recombinant cystic fibrosis transmembrane conductance regulator in epithelial cells and its functional reconstitution into cells in vitro by J Marshall, S Fang, L S Ostedgaard, C R O'Riordan, D Ferrara, J F Amara, H Hoppe, 4th, R K Scheule, M J Welsh, A E Smith

“…We have generated several clones of Chinese hamster ovary, mouse epitheloid C127, and pig kidney epithelial LLCPK1 cells producing high levels of functional…”
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Airway delivery of cationic lipid: DNA complexes for cystic fibrosis by Cheng, S.H, Scheule, R.K

“…Cationic lipids are under active consideration as gene delivery vehicles for cystic fibrosis. Initial studies have shown cationic lipids to be effective agents…”
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Preparation of radiolabeled bioactive asbestos fibers by Tewson, T J, Francsechini, M P, Scheule, R K, Holian, A

“…We have developed an efficient procedure to radiolabel asbestos fibers while retaining the bioactivity of the fibers. The fibers are labeled with 68Ge. The…”
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What is a Successful Reconstitution of a Membrane Glycoprotein that Lacks an Enzymatic Activity? by Gaffney, B.J., Scheule, R.K.

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