gene therapy for cardiovascular disease

... 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 ... was the first recipient of liver-directed gene therapy She was homozygous for a mutation in the LDL receptor gene, result- 162 GENE THERAPY FOR LIVER DISEASE ing in the expression of a nonfunctional ... damages such as motor 168 GENE THERAPY FOR LIVER DISEASE FIGURE 7.6 Gene therapy for a1-antitrypsin (AAT) deficiency A plasmid that contains the full-length human AAT gene is encapsulated in small...

Ngày tải lên: 10/04/2014, 22:11

... 184 GENE THERAPY IN CARDIOVASCULAR DISEASE GENETIC MANIPULATION OF CARDIOVASCULAR TISSUE Modulating Gene Expression in Cardiovascular Tissue Gene therapy can be defined as any manipulation of gene ... READINGS Cardiovascular Gene Therapy Allen, MD Myocardial protection: Is there a role for gene therapy Ann Thorac Surg 68:1924–1928, 1999 Amant C, Berthou L, Walsh K Angiogenesis and gene therapy ... the gene, preventing the translation into protein Another form of gene blockade is the use FIGURE 8.1 Gene therapy strategies See color insert (A) Gene transfer involves delivery of an entire gene, ...

Ngày tải lên: 10/04/2014, 22:11

... of aspirin for primary prevention Page of The aims of this project are: Assess the current use of aspirin therapy for primary and secondary cardiovascular disease prevention in six General Internal ... appropriate use of aspirin for cardiovascular disease prevention for academic detailing to clinicians, a point-of-care decision support aid for clinicians, and a patient activation form; and Test the ... of evidence for low-dose aspirin for primary ASA = Low-dose aspirin Figure CONSORT diagram of the study Page of prevention of cardiovascular disease (CVD) The patient activation form, shown in...

Ngày tải lên: 10/08/2014, 11:20

... with an exposure time of 50 ms for DAPI and 500 ms for EGFP (1 s for negative expression) Page of (page number not for citation purposes) Genetic Vaccines and Therapy 2009, 7:9 http://www.gvt-journal.com/content/7/1/9 ...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

... end-stage renal failure [8] The prevalence of Fabry disease is estimated at : 40 000 for the classic form The incidence of the variant form of Fabry disease was found to be higher Screening of various ... to Fabry disease, small molecules capable of specifically rescuing misfolded enzyme proteins have been identified for Gaucher disease [20,21], Tay-Sachs and Sandhoff disease [22] (details for Gaucher ... weightÆday)1 for weeks [47] No apparent toxic effects were observed in transgenic mice treated with DGJ for 140 days, indicating that DGJ is well tolerated in mice ASSC therapy for Fabry disease in...

Ngày tải lên: 30/03/2014, 03:20

... 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

... 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

... Nanogene Therapy for Lung Disease with those from CIN-treated mice The gene array analysis was repeated once with a similar expression profile Ribonuclease Protection Assay for Cytokine Gene ... experiment Kong et al, Chitosan Interferon-c Nanogene Therapy for Lung Disease 103 Figure Chitosan interferon-c nanogene (CIN) treatment induces changes in gene expression in lung dendritic cells A, ... facilitate gene delivery to mucosa- and bronchus-associated lymphoid tissue Chitosan, therefore, appears to possess all of the attributes of an ideal gene delivery agent for effective nonviral gene...

Ngày tải lên: 08/08/2014, 21:20

... follow up period Cardiovascular disease and risk factors for it CVD was defined as a verified medical history of coronary, cerebral or peripheral arterial disease Coronary artery disease included ... factors for CVD was done for two reasons Firstly, because these risk factors could be overor under-represented in certain DMARD groups and, therefore, falsely influence the cardiovascular risk for ... correction for known cardiovascular risk factors was done to explore possible pathways by which the investigated DMARDs can influence cardiovascular risk; for example, a DMARD could increase the cardiovascular...

Ngày tải lên: 09/08/2014, 08:22

... established disease? The emerging paradigm for the treatment of chronic inflammatory diseases such as RA is early aggressive therapy with tight control of disease activity aimed at robust suppression ... etanercept monotherapy with the response to cotherapy with methotrexate or another disease- modifying antirheumatic drug in patients with rheumatoid arthritis: results from the British Society for Rheumatology ... al.: Feasibility of allogeneic hematopoietic stem cell transplantation for autoimmune disease: position statement from a National Institute of Allergy and Infectious Diseases and National Cancer...

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, ... not yet a clinically effective treatment for CF lung disease primary research Gene therapy will probably prove to be most beneficial if given very early, before the onset of established infection...

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

... (PI3K) isoforms may also Ó FEBS 2002 Cardiovascular receptor crosstalk (Eur J Biochem 269) 4717 Fig Regulation of short and long-term signalling in normal cardiovascular physiology and disease ... individual environmental requirements Implications of receptor crosstalk for cardiac adaptation to diseases Cardiovascular diseases fall broadly into three main categories The first group comprises ... practical significance for human disease conditions The second category of cardiac diseases comprises disorders, such as left ventricular dysfunction from valvular heart diseases or hypertension...

Ngày tải lên: 21/02/2014, 15:20

... 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 ... SFG-F/S-IRES-tk Retrovirus pNFnB-TK Plasmid 156 Virotherapy Virotherapy Transcriptional targeting Delivery of p21 or p53 HSV-TK suicide gene therapy HSV-TK suicide gene therapy Reference Phase I/II/III clinical ... 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

... 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

... single or combined immune therapy modalities be ineffective, combining active or passive immunotherapy approaches with other gene therapy approaches may come to fruition For instance, our group ... tumors identify potential targets for immunotherapy J Neurooncol 2008, 88:65-76 Bao L, Sun Q, Lucas KG: Rapid generation of CMV pp65-specific T cells for immunotherapy J Immunother 2007, 30:557-61 ... response to therapy such as age, maximal resection or minimal/stable residual disease at the start of vaccine therapy, and concurrent administration of chemotherapeutics [23,24,46-51] For valid...

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