molecular medicine and gene therapy an introduction

... ribozymes and dominant negative molecules with limited success in animal models of infection and disease SUGGESTED READINGS General Branch AD A hitchhiker’s guide to antisense and nonantisense ... normal gene for AAT is designated M and accounts for 95% of alleles in the caucasian American population The most common mutants, called Z and S occur with an allelic frequency of to 2% and to ... vivo gene therapy for FH In these studies, hepatocytes were harvested, genetically modified ex vivo with retroviruses that contained an intact LDL receptor gene, and transplanted back into the animal...

Ngày tải lên: 10/04/2014, 22:11

... practical work and drafted the manuscript WW participated in the practical work and discussions JR designed this study and edited the manuscript All authors read and approved the final manuscript ... neutralizing antibody response and transgene expression after AAV1 vector re-administration in mice Gene Ther 2008, 15(1):54-60 Publish with Bio Med Central and every scientist can read your ... citation purposes) Genetic Vaccines and Therapy 2009, 7:9 Acknowledgements http://www.gvt-journal.com/content/7/1/9 18 We thank Günter Schwarz (Köln) for providing cPMP and Sebastian Kügler (Göttingen)...

Ngày tải lên: 14/08/2014, 19:22

... A156V, L166V, and R356W), and two mutations present in both variant and classic patients (A97V and R301Q) were efficiently purified from transfected COS-7 cells, and their enzymatic and biochemical ... Tay-Sachs and Sandhoff disease [22] (details for Gaucher and Tay-Sachs ⁄ Sandhoff diseases are reviewed separately), GM1-gangliosidosis [23], and retinitis pigmentosa 17 [24] Small molecular antagonists ... system and glucosidases I and II promote refolding, whereas ASSC therapy for Fabry disease ER a-mannosidases and the ER degradation enhancing a-mannosidase I-like protein are involved in retrotranslocation...

Ngày tải lên: 30/03/2014, 03:20

... are used in the gene therapy for congenital and acquired immunodeficiencies, malignancies, and GVHD SUGGESTED READINGS Gene Transfer Kiem HP, Andrews RG, Morris J Improved gene transfer into baboon ... autologous stroma As many as to 20% of marrow CFU were shown to be neomycin-resistant between and 18 months posttransplantation, suggesting effective transduction and ongoing transgene expression This ... stimulated peripheral blood, and cord blood and can be cultured and transduced ex vivo before intravenous reinfusion These features have made HSCs an ideal target for gene therapy of a wide variety...

Ngày tải lên: 10/04/2014, 22:10

... gene therapy Ann Thorac Surg 68:1924–1928, 1999 Amant C, Berthou L, Walsh K Angiogenesis and gene therapy in man: Dream or reality Drugs 59(Spec No 33–36), 1999 Ponder KP Systemic gene therapy ... by in vivo gene transfer of endothelial cell–nitric oxide synthase (ecNOS) in balloon-injured rat carotid arteries See color insert GENE THERAPY FOR ANGIOGENESIS Angiogenesis and Angiogenic Factors ... percutaneous catheter-based approach (Fig 8.3) A number of phase I safety studies have been reported in which angiogenic GENE THERAPY FOR ANGIOGENESIS 191 FIGURE 8.3 Combined gene transfer and transmyocardial...

Ngày tải lên: 10/04/2014, 22:11

... binding and consequently inhibit translation An additional clinical protocol for AIDS gene therapy uses retroviral-mediated gene transfer to deliver antisense TAR and RevTD genes to syngeneic ... In Dickson G (Ed.), Molecular and Cell Biology of Human Gene Therapeutics Chapman and Hall, London, 1995, pp 195–236 VandenDriessche T, Chuah MKL, Morgan RA Gene therapy for acquired immune deficiency ... improvements in the gene transfer technology and enhanced gene expression may eventually make HSCs viable candidates for use in gene therapy Due to the inefficiency of gene transfer into HSCs, investigators...

Ngày tải lên: 10/04/2014, 22:11

... (Invitrogen, San Diego, CA), and complexed with chitosan, as described previously.45 Briefly, plasmids in 25 mM Na2SO4 and chitosan (Vanson, Kong et al, Chitosan Interferon-c Nanogene Therapy for ... Chitosan Nanoparticle– Delivered Plasmid To determine that pDNAs can be transported in chitosan nanoparticles and expressed in the lung epithelium, pEGFP was complexed with chitosan nanoparticles and ... wasp venom immunotherapy33 and decreases the production of sulfidoleukotrienes by human PBLs.34 IFN-c also downregulates transforming growth factor b and procollagen I and III and decreases fibrosis...

Ngày tải lên: 08/08/2014, 21:20

... to normalize and returned to pretreatment levels before relapse In SLE patients, antinuclear antibody and antidouble-stranded DNA antibodies disappeared in many patients after HSCT and returned ... cytokineindependent and antigen-independent naturally occurring population, and another cell type that is recruited by the cognate antigen and immunoregulatory cytokines and thus named adaptive ... Cyclophosphamide or Transplantation’ trial – compares safety and efficacy of a different transplant regimen versus intravenous pulse therapy cyclophosphamide The protocols of the ASTIS and ‘Scleroderma:...

Ngày tải lên: 09/08/2014, 10:20

... but will observe the ‘CVD and on aspirin’ and ‘CVD but not on aspirin’ groups to monitor for any change, particularly an unwanted drop in use of anti-platelet aspirin therapy in patients with ... progression of arterial disease and diabetes (POPADAD) trial: factorial randomised placebo controlled trial of aspirin and antioxidants in patients with diabetes and asymptomatic peripheral arterial ... American Stroke Association [17], and the American Diabetes Association [19], and is prescribed for primary prevention by physicians worldwide There is often a disparity between current evidence and...

Ngày tải lên: 10/08/2014, 11:20

... devoid of glands A central question for CF gene therapy is which cell type and which region (large or small airways) to target Although ciliated superficial epithelium is abundant and displays ... successful CF gene therapy have now been clearly defined, and gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy 12 13 14 15 16 References Riordan JR, ... increasingly being investigated, and cationic liposomes and polymers are increasingly being coupled to peptides, sugars and viral particles to try to increase gene transfer efficiency The vexing...

Ngày tải lên: 12/08/2014, 18:20

... coverage Epo gene transfer has already been tested on normal animals and on anaemia animal models such as β-thalassemia and chronic renal failure models To this end, various gene transfer strategies ... Genetic Vaccines and Therapy 2008, 6:10 Statistical analysis Analysis of variance (ANOVA) and Fisher PLSD were used 80 75 70 65 Hematocrit (%) Sample collection, measurement and assay Blood samples ... protocol could be applied to another anaemic context and extend to a wide variety of gene therapy applications using many candidate therapeutic genes such as growth factor genes Competing interests...

Ngày tải lên: 14/08/2014, 19:22

... (nuclear antibody (ANA), smooth muscle antibody (SMA), antibody to the liver/kidney microsome type 1, and anti-mitochondrial antibody), and a standard metabolic/inborn error panel (lactate/pyruvate ... syndrome In the posttransplant period, she was diagnosed with recurrent NASH at months, stage fibrosis at years and months, and stage fibrosis at years and months after liver transplantation She Table ... aminotransferase levels;(2) average daily ethanol consumption of less than 10 g; and (3) appropriate exclusion of other liver diseases based on standard clinical, laboratory, imaging and/ or liver...

Ngày tải lên: 22/03/2014, 10:20

... suicide gene therapy Antiangiogenic gene therapy Virotherapy Virotherapy Measles virus Virotherapy Measles and mumps viruses Virotherapy Adenovirus AAV Delivery of p73 HSV-TK suicide gene therapy ... trial In vitro and in vivo Adenovirus Ad5/3-D24 Main result In vitro in vivo In vitro in vivo In vitro in vivo In vitro in vivo In vitro in vivo and and and and and Killing of ovarian cancer cells, ... in vitro and in vivo by incorporating an integrin-binding RGD-4C motif into the adenoviral fiber [41,42], and a trial is forthcoming Antiangiogenic Gene Therapy Antiangiogenic gene transfer inhibits...

Ngày tải lên: 22/03/2014, 11:20

... 2011;68(4):183-8 65 van Vliet M, von Rosenstiel IA, Schindhelm RK, Brandjes DP, Beijnen JH, Diamant M The association of elevated alanine aminotransferase and the metabolic syndrome in an overweight and obese ... NAFLD and its related complications can be expected in the near future Studying the underlying mechanisms and broadening efforts to prevent and control the main universal risk factors, obesity and ... Obesity and Air Pollution diagnosis, and pathogenesis of pediatric NAFLD, as well as the possible associations with obesity and air pollution, which are the adverse effects of urbanization and globalization...

Ngày tải lên: 29/03/2014, 18:20

... P., Subramanian, A., Gwazdauskas, F C., Johnson, J L., and Velander, W H (1995) Transgenesis in mice by cytoplasmic injection of polylysine/DNA mixtures Transgenic Res 4, 353–360 Wu, G Y and Wu, ... Subheading 3.1.8 and Notes and 3.3 GPC of pDMAEMA Homo- and Copolymers This instruction is intended for the molecular weight determination of pDMAEMA and its copolymers Prepare eluent: 0.7 M NaNO3, 0.1 ... Dextran 50,000 Dextran 750,000 D(+)-Maltose Dextran 1000 Dextran 80,000 Dextran 800,000 Maltotriose Dextran 5000 Dextran 150,000 Maltotetraose Dextran 12,000 Dextran 270,000 Maltopentose Dextran...

Ngày tải lên: 11/04/2014, 09:59

... Sciences, The University of Auckland, Auckland, New Zealand BIAO LI • Center for Human Molecular Genetics, Munroe-Meyer Institute; and Department of Cell Biology and Anatomy, University of Nebraska ... for Gene Transfer into the Rat Brain: Evaluation of Gene Transfer Efficiency, Toxicity, and Inflammatory and Immune Reactions Andres Hurtado-Lorenzo, Anne David, Clare Thomas, Maria G Castro, and ... O’Neill and Andrew Bowie, 2001 75 Lung Cancer: Volume 2, Diagnostic and Therapeutic Methods and Reviews, edited by Barbara Driscoll, 2003 74 Lung Cancer: Volume 1, Molecular Pathology Methods and...

Ngày tải lên: 11/04/2014, 10:39

... African-American and European-American breast cancer patients PLoS One 2009, 4:e4531 18 Tsai MH, Cook JA, Chandramouli GV, DeGraff W, Yan H, Zhao S, Coleman CN, Mitchell JB, Chuang EY: Gene expression ... Cancer, The Verona Hospital Concern and The University of Verona, Verona, Italy 3Infectious Disease and Immunogenetics Section (IDIS), Department of Transfusion Medicine, and Center for Human ... plasmid for transfections and carried out silencing experiments MC, SC and SE performed western blot analysis, part of silencing experiments and helped sketch the manuscript JAC coordinated and GDP...

Ngày tải lên: 18/06/2014, 16:20

... IDH1, EGFR, and NF1 Cancer Cell 2010, 17:98-110 Huse JT, Holland EC: Targeting brain cancer: advances in the molecular pathology of malignant glioma and medulloblastoma Nat Rev Cancer 2010, 10:319-31 ... gathered references and generated the tables and figure MRJ, RMP, LML provided information to shape the manuscript content and discussion All authors have read and approved the final manuscript Received: ... from tetanus toxoid has shown promise in mouse glioma studies, and in an in vitro study using human glioma tissue and autologous PBMC [25,26] Last, at Massachusetts General/Dana Farber Cancer Institute...

Ngày tải lên: 18/06/2014, 16:20

... the experiments and wrote the paper draft; LS conceived, instructed the experiments and revised the paper; SG and XX performed experiments and analyzed data; ZX and ZP collected and validated tissue ... PCFC-g-PEI, FA-PEAs and HPEI as a gene delivery vector respectively And figure B, D and F represented the transfection image with 25-kD PEI and 2-kD PEI as gene vectors Figure a-f showed the transfection ... model and VHL -gene therapy mediated by PEI system The following animal experiments were in compliance with all regulatory guidelines and were approved by the Institutional Animal Care and Use...

Ngày tải lên: 18/06/2014, 19:20