gene therapy for vascular diseases

... with an exposure time of 50 ms for DAPI and 500 ms for EGFP (1 s for negative expression) Page of (page number not for citation purposes) Genetic Vaccines and Therapy 2009, 7:9 http://www.gvt-journal.com/content/7/1/9 ... 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) for ... borderline result and indicates a range for the minimal dosage required for abolishing the MoCo deficiency phenotype With a maximum body weight of 40 g for the mice used here this would correspond...

Ngày tải lên: 14/08/2014, 19:22

... PROGENITOR CELLS AS TARGETS FOR GENE THERAPY 143 144 GENE THERAPY FOR HEMATOLOGICAL DISORDERS restoration of cellular and humoral immunity were documented after gene therapy Data showed a surprisingly ... otherwise standard MuLV vectors for gene therapy has not been successful Beyond these HEMATOPOIETIC STEM AND PROGENITOR CELLS AS TARGETS FOR GENE THERAPY 137 efforts, there are obviously major ... In vivo gene transfer is most appropriate for target cells that cannot REQUIREMENTS FOR GENE TRANSFER INTO HEMATOPOIETIC CELLS 135 TABLE 6.1 Relevant Targets and Applications for Gene Therapy...

Ngày tải lên: 10/04/2014, 22:10

... hand, it is necessary for any vehicle used for in 156 GENE THERAPY FOR LIVER DISEASE vivo hepatic gene therapy to reach the liver efficiently For systemic application, the gene vectors are ideally ... attempted so far GENERAL PRINCIPLES FOR HEPATIC GENE THERAPY There are two basic approaches for gene transfer into hepatocytes: ex vivo and in vivo strategies (Fig 7.1) Ex vivo therapy requires ... expression of the therapeutic gene. Alternatively, different viral vectors as well as liposomes can be used for ex vivo gene transfer GENERAL PRINCIPLES FOR HEPATIC GENE THERAPY 155 Collagenase treatment...

Ngày tải lên: 10/04/2014, 22:11

... competes for an essential substrate or co-factor that is available in limiting amounts, or, for proteins that form multimeric complexes, the 270 GENE THERAPY FOR HIV INFECTION TABLE 11.1 Gene Therapy ... gene expression Thus, the antisense gene expression must be quantatively higher than the levels of HIV-1 gene expression for an antisense gene therapy strategy to be effective Standard gene therapy ... levels in HSCs Thus, retroviral-mediated gene transfer is currently the optimal gene transfer system available for use in HIV-1 gene therapy The 282 GENE THERAPY FOR HIV INFECTION limitations of retroviral...

Ngày tải lên: 10/04/2014, 22:11

... R, Krause D, Keating A: Clarification of the nomenclature for MSC: the International Society for Cellular Therapy position statement Cytotherapy 2005, 7:393-395 Colter DC, Sekiya I, Prockop DJ: ... pathogenic clones Now that technologies for cell purification and protocols for expanding specific subsets are more advanced, there are opportunities for achieving immune homeostasis by infusion ... we review and discuss the current and future prospects for such cell-based therapies Hematopoietic stem cell transplantation for autoimmune diseases Hematopoietic stem cell transplantation (HSCT)...

Ngày tải lên: 09/08/2014, 10:20

... to be answered for 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 ... models that successful gene therapy for cystic fibrosis requires only partial gene correction Gene Ther 1996, 3:797–801 28 Hyde SC, Southern KW, Gileadi U, Fitzjohn EM, Mofford KA, Waddell BE, ... has dogged the recent media view of gene therapy Gene therapy is clearly at the typical stage of new drug development where considerable and unglamorous effort needs to be expended to move from...

Ngày tải lên: 12/08/2014, 18:20

... Therefore, in the particular case of Epo, an accurate level of transgene expression is required for safety reasons Temporal control systems of transgene expression have already been used in gene therapy ... erythropoietin gene therapy for patients with chronic renal failure Blood 2005, 106(7):2280-2286 Osada S, Ebihara I, Setoguchi Y, Takahashi H, Tomino Y, Koide H: Gene therapy for renal anemia ... square) Epo-plasmid doses for the other groups Electrotransfer was performed at day 0, 34, 112 and 215 for the three groups One additional electrotransfer was performed at day 77 for the μg group Arrows...

Ngày tải lên: 14/08/2014, 19:22

... angiographic restenosis was associated with a lower rate of TLR (3.5% for SESs vs 18.5% for BMSs; 3.3% for PESs vs 12.2% for BMSs) More recently, the benefits of DESs have been confirmed in studies ... deposits the fibers on the surface, where the fibers deposit to form a thin, uniform mesh Electrospinning generates continuous, uniformed and long fibers, which have diameters down to nano-scale ... A) 35% DMF, B) 40% DMF, C) 45% DMF, D) 50% DMF, E) 15% Chloroform/Methanol (70:30), F) 20% Chloroform/Methanol (70:30), G) 25% Chloroform/Methanol (70:30), H) 15% HFIP and I) 20% HFIP solutions...

Ngày tải lên: 14/09/2015, 08:41

... 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 ... is forthcoming Antiangiogenic Gene Therapy Antiangiogenic gene transfer inhibits formation of neovasculature required for tumor growth and may also act by collapsing immature tumor-associated vascular ... phase I trial [36] Molecular Chemotherapy Molecular chemotherapy (a.k.a suicide gene therapy) is a strategy based on delivery of genes encoding a prodrug- MOLECULAR THERAPY Vol 14, No 2, August 2006...

Ngày tải lên: 22/03/2014, 11:20

... vectors for gene therapy formed by self-assembly of DNA with synthetic block co-polymers Hum Gene Ther 7, 2123–2133 11 Kabanov, A V and Kabanov, V A (1995) DNA complexes with polycations for the ... Nonviral Gene Delivery Gert W Bos, Daan J A Crommelin, and Wim E Hennink Introduction The aim of gene therapy is to treat inherited or acquired genetic deficiencies (e.g., cystic fibrosis) or viral diseases ... refs 8–10 for typical results) The protocol can be used for all adherent cell types For suspension type cells, cells need to be spun down before changing the medium at a speed suitable for the...

Ngày tải lên: 11/04/2014, 09:59

... DAVIDSON • Program in Gene Therapy, Departments of Internal Medicine, Neurology, Physiology & Biophysics, and Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases, University ... vectors for gene transfer and gene therapy I would like to thank the many chapter authors for their contributions They are all experts in various aspects of viral vectors, and I appreciate their efforts ... Type 1-Based Vectors for Gene Delivery to Human Hematopoietic Stem Cells Ali Ramezani and Robert G Hawley 467 Semliki Forest Viral Vectors for Gene Transfer Jarmo Wahlfors and Richard A Morgan...

Ngày tải lên: 11/04/2014, 10:39

... for autologous adipose cell therapy, such as Cytori's Celution™ system [16] and Tissue Genesis' TGI 1000™ platform [17], which are presently entering clinical trials Unfortunately, since the majority ... precursors in vascular repair and lesion formation J Cell Mol Med 2005, 9:557-568 Miranville A, Heeschen C, Sengenes C, Curat CA, Busse R, Bouloumie A: Improvement of postnatal neovascularization ... Although these are generalizations, an initial insult either by foreign microorganisms, or other means, causes tissue damage and activation of innate immunity, which under proper genetic background...

Ngày tải lên: 18/06/2014, 15:20

... important for the resistance to possible introduction of genes using viral vectors or for the resistance to oncolytic gene therapy We believe that this finding can be of crucial interest for the ... viral gene therapy approaches Adenoviruses and Adeno-Associated viruses are used to deliver genes to tumor cells with the goal of modifying the phenotype, as for example, by introducing suicide genes ... primers: Ad type forward primer 5’-AACCGAAGGCTGCATTCACT, reverse primer 5’-ACCGCACAGGGTCTTAATAGAG Following denaturation at 96°C for 10 min, cycling conditions were 96°C for 15s, 60°C for for 40 cycles...

Ngày tải lên: 18/06/2014, 16:20

... as follows: first cycle at 95°C for min, and then 30 cycles at 94°C for 45 s, 54°C for min, 72°C for and a final extension cycle of 72°C for The house-keeping gene b-actin was taken as a loading ... DNA for gene therapy [9,14,15] For example, a liner PEI-cholesterol conjugation was encapsulated with interleukin-12 to treat RCC mice intravenously, which was demonstrated to be effective for ... weight, and the gene therapy effects on RCC model mice were obvious by using FA-PEAs:pVHL complexes to treat tumor Therefore, FA-PEAs may be a potential gene transfer system to carry VHL gene to treat...

Ngày tải lên: 18/06/2014, 19:20

... as follows: first cycle at 95°C for min, and then 30 cycles at 94°C for 45 s, 54°C for min, 72°C for and a final extension cycle of 72°C for The house-keeping gene b-actin was taken as a loading ... DNA for gene therapy [9,14,15] For example, a liner PEI-cholesterol conjugation was encapsulated with interleukin-12 to treat RCC mice intravenously, which was demonstrated to be effective for ... weight, and the gene therapy effects on RCC model mice were obvious by using FA-PEAs:pVHL complexes to treat tumor Therefore, FA-PEAs may be a potential gene transfer system to carry VHL gene to treat...

Ngày tải lên: 20/06/2014, 03:20

... replicate experiments performed for each tissue The striking finding was that statistically significant variable gene expression was detected for 3.3%, 1.9%, and 0.8% of the genes in the kidney, ... between subset-specific and donor-specific changes in gene expression We therefore decided to analyze gene expression in Th1 and Th2 cells generated from three additional donors and to analyze ... interest for pharmacological intervention in chronic inflammatory diseases, we decided to analyze gene expression profiles of human Th1 and Th2 cells Polyclonal human Th1 and Th2 cells were generated...

Ngày tải lên: 09/08/2014, 01:21

... number not for citation purposes) Arthritis Research & Therapy Vol 11 No Evans et al Applications in rheumatic diseases Rheumatoid arthritis Local therapy Interest in applying gene therapy to ... [90] as the transgene products Because the salivary gland has an exocrine function, it can also be used as a site of gene transfer for systemic delivery purposes [91] Gene therapy for Sjögren syndrome ... adenoviral gene transfer Mol Genet Metab 2003, 80: 148-158 Wilson JM: Gendicine: the first commercial gene therapy product Hum Gene Ther 2005, 16:1014-1015 Page of 12 (page number not for citation...

Ngày tải lên: 09/08/2014, 01:22

... and thus the former is the vector of choice for future gene therapy trials The number of gene therapy trials in RA is in sharp contrast to the more than 600 clinical trials of gene therapy in patients ... safety of local gene therapy Gene therapy in RA is still in its adolescence, and more clinical studies must be initiated if it is to mature It is, however, imperative that gene therapy studies ... Sacchi A: Wild-type p53 gene transfer is not detrimental to normal cells in vivo: implications for tumor gene therapy Oncogene 2004, 23:418-425 Kruyt FA, Curiel DT: Toward a new generation of conditionally...

Ngày tải lên: 09/08/2014, 01:24

... examined with the use of fluorescence microscopy for PKH26 dye The sections were stained for hematoxylin and eosin Immunohistochemistry was performed for asarcomeric actin, von Willebrand factor ... Kit (Applied Biosystems, Foster City, CA, USA) was performed for cDNA synthesis The cycling parameters were 25°C for 10 minutes and 37°C for hours Page of 10 Real time RT-PCR analyses were used ... were washed in PBS for times, labeled with a fluorescent dye using PKH26, before suspended in 0.1 mL of culture medium (without serum) at a concentration of × 10 /mL cells for transplantation...

Ngày tải lên: 10/08/2014, 09:21

... Thus, CD34 progenitor cells are an essential ingredient for HIV gene therapy In view of the need for CD34 cells for HIV gene therapy as well as for other hematopoietic disorders, if one can produce ... lineages thus paving the way for utilization of these cells for hematopoietic cell therapy [20,27-29] For the effective utilization of hES-CD34 cells for HIV gene therapy, a number of parameters ... Stem Cell-based Gene Therapy for HIV/AIDS Anticancer Res 2003, 23:1997-2005 Michienzi A, Castanotto D, Lee N, Li S, Zaia JA, Rossi JJ: RNA-mediated inhibition of HIV in a gene therapy setting...

Ngày tải lên: 13/08/2014, 09:21