Search Results - "Howe, S J"

Persistent episomal transgene expression in liver following delivery of a scaffold matrix attachment region containing non-viral vector by Argyros, O, Wong, S P, Niceta, M, Waddington, S N, Howe, S J, Coutelle, C, Miller, A D, Harbottle, R P

“…An ideal gene therapy vector should enable persistent transgene expression without limitations of safety and reproducibility. Here we report the development of…”
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Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors by Rahim, A A, Wong, A M S, Howe, S J, Buckley, S M K, Acosta-Saltos, A D, Elston, K E, Ward, N J, Philpott, N J, Cooper, J D, Anderson, P N, Waddington, S N, Thrasher, A J, Raivich, G

“…Non-integrating lentiviral vectors show considerable promise for gene therapy applications as they persist as long-term episomes in non-dividing cells and…”
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Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34 + cells : implications for leukemogenesis in gene therapy by PIKE-OVERZET, K, DE RIDDER, D, VAN DONGEN, J. J. M, STAAL, F. J. T, WEERKAMP, F, BAERT, M. R. M, VERSTEGEN, M. M. A, BRUGMAN, M. H, HOWE, S. J, REINDERS, M. J. T, THRASHER, A. J, WAGEMAKER, G

“…The occurrence of leukemia in a gene therapy trial for SCID-X1 has highlighted insertional mutagenesis as an adverse effect. Although retroviral integration…”
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Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression by Buckley, S M K, Howe, S J, Sheard, V, Ward, N J, Coutelle, C, Thrasher, A J, Waddington, S N, McKay, T R

“…Gene transfer for cystic fibrosis (CF) airway disease has been hampered by the lung's innate refractivity to pathogen infection. We hypothesized that early…”
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Correction of SCID-X1 using an enhancerless Vav promoter by Almarza, E, Zhang, F, Santilli, G, Blundell, M P, Howe, S J, Thornhill, S I, Bueren, J A, Thrasher, A J

“…The efficacy of gene therapy for the treatment of inherited immunodeficiency has been highlighted in recent clinical trials, although in some cases complicated…”
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Haematopoietic repopulating activity in human cord blood CD133 quiescent cells by Boxall, S A, Cook, G P, Pearce, D, Bonnet, D, El-Sherbiny, Y M, Blundell, M P, Howe, S J, Leek, J P, Markham, A F, de Wynter, E A

“…We have demonstrated previously that cord blood CD133 + cells isolated in the G 0 phase of the cell cycle are highly enriched for haematopoietic stem cell…”
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Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector by LAURIE, K. L, BLUNDELL, M. P, BAXENDALE, H. E, HOWE, S. J, SINCLAIR, J, QASIM, W, BRUNSBERG, U, THRASHER, A. J, HOLMDAHL, R, GUSTAFSSON, K

“…The expression of genes specifically in B cells is of great interest in both experimental immunology as well as in future clinical gene therapy. We have…”
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Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis by Banushi, Blerida, Forneris, Federico, Straatman-Iwanowska, Anna, Strange, Adam, Lyne, Anne-Marie, Rogerson, Clare, Burden, Jemima J., Heywood, Wendy E., Hanley, Joanna, Doykov, Ivan, Straatman, Kornelis R., Smith, Holly, Bem, Danai, Kriston-Vizi, Janos, Ariceta, Gema, Risteli, Maija, Wang, Chunguang, Ardill, Rosalyn E., Zaniew, Marcin, Latka-Grot, Julita, Waddington, Simon N., Howe, S. J., Ferraro, Francesco, Gjinovci, Asllan, Lawrence, Scott, Marsh, Mark, Girolami, Mark, Bozec, Laurent, Mills, Kevin, Gissen, Paul

“…Post-translational modifications are necessary for collagen precursor molecules (procollagens) to acquire final shape and function. However, the mechanism and…”
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Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency by Abdul-Razak, H. H., Rocca, C. J., Howe, S. J., Alonso-Ferrero, M. E., Wang, J., Gabriel, R., Bartholomae, C. C., Gan, C. H. V., Garín, M. I., Roberts, A., Blundell, M. P., Prakash, V., Molina-Estevez, F. J., Pantoglou, J., Guenechea, G., Holmes, M. C., Gregory, P. D., Kinnon, C., von Kalle, C., Schmidt, M., Bueren, J. A., Thrasher, A. J., Yáñez-Muñoz, R. J.

“…Genome editing is the introduction of directed modifications in the genome, a process boosted to therapeutic levels by designer nucleases. Building on the…”
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Lentiviral labeling of mouse and human enteric nervous system stem cells for regenerative medicine studies by Natarajan, D., Cooper, J., Choudhury, S., Delalande, J.‐M., McCann, C., Howe, S. J., Thapar, N., Burns, A. J.

“…Background Reliable methods of labeling human enteric nervous system (ENS) stem cells for use in novel cell replacement therapies for enteric neuropathies are…”
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Targeted expression of human folylpolyglutamate synthase for selective enhancement of methotrexate chemotherapy in osteosarcoma cells by Bienemann, K, Staege, M S, Howe, S J, Sena-Esteves, M, Hanenberg, H, Kramm, C M

“…The antifolate methotrexate (MTX) is an important chemotherapeutic agent for treatment of osteosarcoma. This drug is converted intracellularly into…”
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Facing Your Fears in autistic youth with co-occurring psychiatric conditions: Reductions in symptoms of anxiety and depression by McMorris, C.A., Turner, K., Johnston, K., Clark, C.A., Howe, S.J., McConnell, M., McFee, K.

“…Autistic youth often experience psychiatric complexity, with up to 70 % of youth experiencing at least one mental health issue, and as many as 41 %…”
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Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo by Schwarzwaelder, Kerstin, Howe, Steven J, Schmidt, Manfred, Brugman, Martijn H, Deichmann, Annette, Glimm, Hanno, Schmidt, Sonja, Prinz, Claudia, Wissler, Manuela, King, Douglas J S, Zhang, Fang, Parsley, Kathryn L, Gilmour, Kimberly C, Sinclair, Joanna, Bayford, Jinhua, Peraj, Rachel, Pike-Overzet, Karin, Staal, Frank J T, de Ridder, Dick, Kinnon, Christine, Abel, Ulrich, Wagemaker, Gerard, Gaspar, H Bobby, Thrasher, Adrian J, von Kalle, Christof

“…We treated 10 children with X-linked SCID (SCID-X1) using gammaretrovirus-mediated gene transfer. Those with sufficient follow-up were found to have recovered…”
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Completely predictable and fully anticipated? Step ups in warrant exercise prices by Garcia-Feijóo, Luis, Howe, John S., Su, Tie

“…Some warrants are issued with a scheduled increase in their exercise price. This increase, referred to as a 'step up' in exercise price, occurs after the…”
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The relationship between plasma concentration of valproic acid and its anticonvulsant and behavioural effects in the rat by Tulloch, I F, Walter, D S, Howe, G M, Howe, S J

“…The relationship between the plasma concentration of valproic acid (VPA) and anticonvulsant or neurotoxic effects was studied in the rat. Anticonvulsant…”
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The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray by Bartolovic, Kerol, Mongkoldhumrongkul, Napachanok, Waddington, Simon N, Jayasinghe, Suwan N, Howe, Steven J

“…The bio-electrospray technique has been recently pioneered to manipulate living, immortalised and primary cells, including a wide range of stem cells. Studies…”
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BRONCHIAL ADENOMA WITHIN THE WALL OF A BRONCHOGENIC CYST. REPORT OF A CASE by GREENFIELD, L J, HOWE, J S

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Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration by Buckley, Suzanne M K, Howe, Steven J, Rahim, Ahad A, Buning, Hildegard, McIntosh, Jenny, Wong, Suet-Ping, Baker, Andrew H, Nathwani, Amit, Thrasher, Adrian J, Coutelle, Charles, McKay, Tristan R, Waddington, Simon N

“…In vivo bioimaging of transgenic luciferase in the lung and nose is an expedient method by which to continually measure expression of this marker gene after…”
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Effective gene therapy with nonintegrating lentiviral vectors by Yáñez-Muñoz, Rafael J, Ali, Robin R, Balaggan, Kamaljit S, MacNeil, Angus, Howe, Steven J, Schmidt, Manfred, Smith, Alexander J, Buch, Prateek, MacLaren, Robert E, Anderson, Patrick N, Barker, Susie E, Duran, Yanai, Bartholomae, Cynthia, von Kalle, Christof, Heckenlively, John R, Kinnon, Christine, Thrasher, Adrian J

“…Retroviral and lentiviral vector integration into host-cell chromosomes carries with it a finite chance of causing insertional mutagenesis. This risk has been…”
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How to integrate risk management and quality assurance by Howe, J S

“…While implementing a coordinated quality assurance/risk management program requires resources, primarily for training, it's hard to match the dual benefits of…”
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