gene therapy for liver disease
gene therapy for liver disease
Ngày tải lên :
10/04/2014, 22:11
... 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 targeted to the liver, ... to liver diseases resulting in liver failure To date, therapy for alcoholic liver disease is the cessation of alcohol consumption and in the case of end-stage liver disease (liver failure) liver ... Canadian woman 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...
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Báo cáo sinh học: "AAV-mediated gene therapy for metabolic diseases: dosage and reapplication studies in the molybdenum cofactor deficiency model" pps
Ngày tải lên :
14/08/2014, 19:22
... with an exposure time of 50 ms for DAPI and 500 ms for EGFP (1 s for negative expression) Page of (page number not for citation purposes) Genetic Vaccines and Therapy 2009, 7:9 http://www.gvt-journal.com/content/7/1/9 ... AAV-MOCS1 for systemic delivery, as compared to × 1010 tu for the intrahepatic injections described previously [5] and above, the results described here indicate a similar efficacy for both application ... of (page number not for citation purposes) Genetic Vaccines and Therapy 2009, 7:9 Acknowledgements http://www.gvt-journal.com/content/7/1/9 18 We thank Günter Schwarz (Köln) for providing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...
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Báo cáo khoa học: Active-site-specific chaperone therapy for Fabry disease Yin and Yang of enzyme inhibitors pptx
Ngày tải lên :
30/03/2014, 03:20
... DGJ for 140 days, indicating that DGJ is well tolerated in mice ASSC therapy for Fabry disease in humans The clinical proof-of-concept for ASSC therapy has been investigated in cardiac Fabry disease ... 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 ... 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...
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gene therapy for hematological disorders
Ngày tải lên :
10/04/2014, 22:10
... 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...
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gene therapy in cardiovascular disease
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 ... influences disease This manipulation is generally achieved via the transfection of foreign deoxyribonucleic acid (DNA) into cells Gene therapy can involve either the delivery of whole, active genes (gene ... 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, ...
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gene therapy for hiv infection
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...
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Báo cáo y học: " Chitosan Interferon-c Nanogene Therapy for Lung Disease: Modulation of T-Cell and Dendritic Cell Immune Responses" pps
Ngày tải lên :
08/08/2014, 21:20
... 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 ... 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,...
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Báo cáo y học: " Cell therapy for autoimmune diseases" ppt
Ngày tải lên :
09/08/2014, 10:20
... 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...
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báo cáo khoa học: "Reconsidering low-dose aspirin therapy for cardiovascular disease: a study protocol for physician and patient behavioral change" pot
Ngày tải lên :
10/08/2014, 11:20
... 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 ... clinical decision support system to consider aspirin therapy for all patients with diabetes mellitus, peripheral artery disease, chronic kidney disease, or a calculated Framingham Risk Score >20%...
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Báo cáo y học: " Taking stock of gene therapy for cystic fibrosis" pdf
Ngày tải lên :
12/08/2014, 18:20
... 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...
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Báo cáo sinh học: "Careful adjustment of Epo non-viral gene therapy for β-thalassemic anaemia treatment" pot
Ngày tải lên :
14/08/2014, 19:22
... 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 ... opposite to viral vector mediated gene delivery This repeated delivery protocol allows careful adjustments to reach the clinical endpoint and feedback for subsequent dose delivery This safe treatment...
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The natural history of non-alcoholic fatty liver disease in children: a follow-up study for up to 20 years pptx
Ngày tải lên :
22/03/2014, 10:20
... of other liver diseases based on standard clinical, laboratory, imaging and/or liver biopsy features Laboratory tests to rule out other liver diseases included a viral hepatitis panel (for hepatitis ... Persistent abnormal liver tests Liver biopsy performed for persistently elevated liver enzymes and done during bariatric surgery – Persistent abnormal liver tests Follow-up before starting treatment ... Follow-up before ursodeoxycholic acid prescribed – Worsening of ALT levels Liver biopsy performed for persistently elevated ALT and done during laparoscopic cholecystectomy – Persistent abnormal liver...
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Gene Transfer Approaches for Gynecological Diseases pot
Ngày tải lên :
22/03/2014, 11:20
... 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...
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Obesity and Air Pollution: Global Risk Factors for Pediatric Non-alco- holic Fatty Liver Disease potx
Ngày tải lên :
29/03/2014, 18:20
... environmental factors on liver 57 diseases Environmental exposure related to toxic waste sites was associated with an increased prevalence of autoimmune liver disease (83, 84) Therefore, increasing attention ... pollution on liver disease in the general population A large population-based study was conducted on 4582 adult participants without viral hepatitis, hemochromatosis, or alcoholic liver disease, ... 2010;1(2):131-7 23 Angulo P Nonalcoholic fatty liver disease N Engl J Med 2002;346(16):1221-31 24 Younossi ZM, Diehl AM, Ong JP Nonalcoholic fatty liver disease: an agenda for clinical research Hepatology...
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nonviral vectors for gene therapy, methods and protocols
Ngày tải lên :
11/04/2014, 09:59
... 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 delivery ... 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 ... (1999) A novel gene delivery system for genetic manipulation of sinusoidal )endothelial cells by triplex DNA technology: evaluation of targetability and ability to stabilize triplex formation Hepatology...
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viral vectors for gene therapy
Ngày tải lên :
11/04/2014, 10:39
... 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 ... 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 ... paper) Gene therapy Laboratory manuals Genetic vectors–Laboratory manuals Transfection–Laboratory manuals Viral genetics–Laboratory manuals I Machida, Curtis A II Series [DNLM: Genetic Vectors Gene...
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Báo cáo hóa học: "Anti-viral state segregates two molecular phenotypes of pancreatic adenocarcinoma: potential relevance for adenoviral gene therapy" pdf
Ngày tải lên :
18/06/2014, 16:20
... 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 ... 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 ... 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...
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Báo cáo hóa học: " Abstract Despite new additions to the standard of care therapy for high grade primary malignant brain tumors, the prognosis for patients with this disease is still poor." ppt
Ngày tải lên :
18/06/2014, 16:20
... [11-14] Delivery of the gene- modified allogeneic T cells given with aldesleukin (IL-2) for newlydiagnosed patients with WHO grade III or IV brain tumors is by convection enhanced delivery (CED) ... 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...
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Báo cáo sinh học: "Comparisons of three polyethyleneimine-derived nanoparticles as a gene therapy delivery system for renal cell carcinoma" doc
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 ... (FA), heparin and so on [4] Furthermore, several PEI-based delivery vehicles have been used to carry DNA for gene therapy [9,14,15] For example, a liner PEI-cholesterol conjugation was encapsulated ... as a gene delivery vehicle Among the total non-viral gene vectors, PEI, with high transfection efficiency, has bright prospects in application [9,10] However, PEI is not fit for keeping on gene...
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