building a better mouse genetically altered mice as models for gene therapy
gene therapy for liver disease
... expression Addition of araAMP araAMP Tk Cells express VZV-TK from AFP promoter Addition of araAMP araATP araAMP Cells die HCC cells araATP Cells grow araAMP araATP araAMP Cells grow Tk araATP Cells die ... molecules AA Nu ol cl C A G U AG G G eu A C B AT T CU C A G C U NA pairing UG AC al D os C som hro mo A A Antisense region I Cleavage CA U AAA UACUCCGUC CGCU GAA A A UG A GG CA U C UCCGC A A U AG AG ... hepatocytes and results in measurable levels of human AAT in mouse plasma, lasting 11 days If a partial hepatectomy is performed h after the intravenous application of the liposomal formulation, AAT plasma...
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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
... lines and an intrahepatic AAV-EGFP injection months after the last AAV-MOCS1 injection Liver sections of animals are shown for each time point Positive control animals (+) received only an AAV-EGFP ... Herati R, Wang B, Cullen K, O'Donnell P, Ellinwood NM, Traas A, Primeau TM, Haskins ME: Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can ... mice were pretreated for 40 days after birth with purified cPMP as described above At day 40, the two animals obtained a single intravenous tail vein injection of 65 μl containing × 109 tu AAV-MOCS1...
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Báo cáo khoa học: Active-site-specific chaperone therapy for Fabry disease Yin and Yang of enzyme inhibitors pptx
... disease: an asymptomatic hemizygote with significant residual a- galactosidase A activity Am J Hum Genet 33, 7 1A Nakao S, Takenaka T, Maeda M, Kodama C, Tanaka A, Tahara M, Yoshida A, Kuriyama M, Hayashibe ... 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 and degradation ... Sakuraba H et al (1995) An atypical variant of Fabry’s disease in men with left ventricular hypertrophy N Engl J Med 333, 288–293 Nakao S, Kodama C, Takenaka T, Tanaka A, Yasumoto Y, Yoshida A, ...
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gene therapy for hematological disorders
... Specific aspects of gene transfer techniques are advantageous for gene therapy approaches when applied to hematological diseases Aspects of ex vivo gene transfer as well as certain gene transfer ... precursors Fanconi anemia (FA) is a hematopoietic genetic disorder that may be an excellent candidate for gene therapy FA is a bone marrow failure syndrome, characterized by physical anomalies, and an ... of hematopoietic cells for several reasons The human ADA gene was cloned in the early 1980s and the small 1.5-kb (cDNA) could easily fit into a retroviral vector along a selectable marker gene...
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gene therapy in cardiovascular disease
... 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 ... these observations have laid the basis for consideration of gene transfer as a therapeutic approach to cardiac disease Additionally, both adenoviral and adenoassociated viral vectors can be delivered ... currently available to a significant number of patients suffering from untreatable 192 GENE THERAPY IN CARDIOVASCULAR DISEASE disease In addition, angiogenic gene therapy may offer an adjunct to traditional...
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gene therapy for hiv infection
... GENE THERAPY APPROACHES 275 Saturation of Tat Protein Decoys Tat Transactivator Tar 5' Decoys Element Viral mRNA 3' Viral mRNA Antisense Nucleic Acids 3' Viral mRNA 5' 5' 5' Degradation by RNase ... the env gene and the core proteins (p6, p9, p17, and p24) which are encoded by the gag gene The pol gene generates the viral-associated reverse transcriptase, integrase, RNase H, and protease enzyme ... cleavage site A distinct advantage of ribozymes over traditional antisense RNA is that they are not consumed during the target cleavage reaction.Therefore, a single ribozyme can inactivate a large...
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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
... L, Farraj NF, Davis SS Chitosan as a novel nasal delivery system for peptide drugs Pharm Res 1994;11:1186–9 20 Miyazaki S, Nakayama A, Oda M, et al Chitosan and sodium alginate based bioadhesive ... Kong et al, Chitosan Interferon-c Nanogene Therapy for Lung Disease 46 Kumar M, Kong X, Behera AK, et al Chitosan IFN-gamma-pDNA nanoparticle (CIN) therapy for allergic asthma Genet Vaccines ... was isolated and assayed with the mouse TranSignal Interferon-inducible Gene Array Kit, as described in Materials and Methods The arrays were scanned and the images analyzed using the ScionImage...
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Báo cáo y học: " Cell therapy for autoimmune diseases" ppt
... Y, Araki H, Kato J, Nakamura K, Kawano Y, Kobune M, Sato T, Miyanishi K, Takayama T, Takahashi M, et al.: Human mesenchymal stem cells xenografted directly to rat liver differentiated into human ... arthritis: analysis of clinical effects, mortality, and transplant related morbidity Ann Rheum Dis 2004, 63: 1318-1326 24 Gratwohl A, Passweg J, Bocelli-Tyndall C, Fassas A, van Laar JM, Farge D, Andolina ... of patients with 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...
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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
... (POPADAD) trial: factorial randomised placebo controlled trial of aspirin and antioxidants in patients with diabetes and asymptomatic peripheral arterial disease BMJ 2008, 337 :a1 840 Ogawa H, Nakayama ... had no associated cardiovascular diagnosis were classified as ‘No CVD but on aspirin’ (N = 4,400) Those who had no documentation of aspirin or other anti-platelet therapy, but who carried a cardiovascular ... 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 Additional File 1, was...
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Báo cáo y học: " Taking stock of gene therapy for cystic fibrosis" pdf
... an important advantage, with a third application having been shown to be as effective as the first in a recent trial of multiple applications of liposome-mediated gene therapy to the nasal epithelium ... duration of expression after a single application is measurable in weeks This does not matter as long as multiple applications are feasible In this regard, cationic liposomes seem to have an ... High KA: Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector Nat Genet 2000, 24:257–261 Authors’ affiliation: Department of Gene Therapy, ...
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Báo cáo sinh học: "Careful adjustment of Epo non-viral gene therapy for β-thalassemic anaemia treatment" pot
... 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 have been ... Looking at individual data, it appears that the hematocrit can be maintained at an almost constant level for each of the treated animal This strategy allows to avoid the deleterious initial hematocrit ... (ESOPE) and validated for clinical use As far as muscle electrotransfer is concerned, at least two clinical trials have been approved and are being conducted in the area of cancer vaccination by...
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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
... (serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase activity, c-glutamyl transferase (GGT), total bilirubin, albumin levels and prothrombin time), fasting ... lipoprotein; M, male The AST/ALT ratio was greater than in 29% of children GGT was elevated in 88% of patients while serum alkaline phosphatase was above the normal value for age and sex in few patients ... Proportion within normal range* (%) Alanine aminotransferase (U/l) Serum aspartate aminotransferase (U/l) Alkaline phosphatase (U/l) c-Glutamytransferase (U/l) Total bilirubin (mg/dl) Albumin (g/dl)...
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Gene Transfer Approaches for Gynecological Diseases pot
... plasmid-based transduction in the context of advanced disease A similar strategy was used in another phase I trial [36] Molecular Chemotherapy Molecular chemotherapy (a. k .a suicide gene therapy) ... ONYX015 has been evaluated in a phase I ovarian cancer trial [60] Treatment resulted in grade abdominal pain and diarrhea in one patient but the maximum tolerated dose was not reached, and the bmaximum ... Phase I/II clinical trial Phase I clinical trial Phase I clinical trial Phase I clinical trial Phase I clinical trial Phase I clinical trial In vitro and in vivo Adenovirus Ad5/3-D24 Main result In...
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Obesity and Air Pollution: Global Risk Factors for Pediatric Non-alco- holic Fatty Liver Disease potx
... Mohammad-Alizadeh AH, Esna-Ashari F, Ardalan G, Hajarizadeh B Non-alcoholic fatty liver disease prevalence among school-aged children and adolescents in Iran and its association with biochemical and ... feasible in large epidemiological studies, surrogate markers such as serum alanine/aspartate aminotransferases (ALT/AST) or ultrasonography are usually used to detect NAFLD (27) The normal range ... polychlorinated biphenyls as well as heavy metals, notably lead and mercury, was associated with unexplained ALT elevation, and increased adjusted ORs for ALT elevation in a dose-dependent manner...
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nonviral vectors for gene therapy, methods and protocols
... cationic amino acids is used as a gene carrier into cells Wyman et al (1) employed a peptide, KALA (WEAKLAKA-LAKA-LAKH-LAKA-LAKA-LKAC-EA), which is derived from the sequence of the N-terminal ... sinusoidal )endothelial cells by triplex DNA technology: evaluation of targetability and ability to stabilize triplex formation Hepatology 30, 29 8A 19 Asayama, S., Nogawa, M., Takei, Y., Akaika, T., ... Japan) Matrix-assisted Laser Desorption Ionization-Time of Flight-Mass Spectra (MALDI TOF-MS) apparatus (Voyager DE STR, PE Biosystems) 2.2 Preparation of Plasmid DNA Plasmid DNA, which contains...
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viral vectors for gene therapy
... therapy Laboratory manuals Genetic vectors–Laboratory manuals Transfection–Laboratory manuals Viral genetics–Laboratory manuals I Machida, Curtis A II Series [DNLM: Genetic Vectors Gene Therapy Gene Transfer ... Alabama at Birmingham, Birmingham, AL MATTHIAS KLUGMANN • Department of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland, Auckland, New Zealand ... Lew, A M., Grob, P., Adema, G J., Ackermann, M., Shortman, K., and Fraefel, C (1999) BAC-VAC, a novel generation of (DNA) vaccines: A bacterial artificial chromosome (BAC) containing a replication-competent,...
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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
... Beverly Handelman(NIH/NIDCR) This work was supported by: Associazione Italiana Ricerca Cancro (AIRC), Milan, Italy (AS) ; Fondazione CariPaRo, Padova, Italy (AS) ; Banco Popolare di Verona (VM); ... supernatant after lysis was collected for testing viral load by real time qPCR AAV infection was performed in Costar black 96 well plates with clear flat bottom (Corning, NY) Luciferase assay was ... P, Kreike B, et al: An interferon-related gene signature for DNA damage resistance is a predictive marker for chemotherapy and radiation for breast cancer Proc Natl Acad Sci USA 2008, 105:18490-18495...
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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
... leukemia patients with residual disease on imatinib mesylate Clin Cancer Res 2010, 16:338-47 Moriai R, Asanuma K, Kobayashi D, Yajima T, Yagihashi A, Yamada M, Watanabe N: Quantitative analysis ... interests The authors declare that they have no competing interests Abbreviations (A) : astrocytoma; (AA): anaplastic astrocytoma; (alloCTL): alloreactive cytotoxic T lymphocytes; (AODG): anaplastic oligodendroglioma; ... major histocompatibility complex; (MAG): mixed anaplastic glioma aka mixed anaplastic oligoastrocytoma; (MG): mixed glioma aka mixed oligoastrocytoma; (MLR): mixed lymphocyte reaction; (mRNA):...
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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
... Sugiyama A, Kume H, Ota S, Kashima T, Tomita K, Kitamura T, Kodama T, Fukayama M, Aburatani H: Identification of Toll-like receptor as a potential therapeutic target in clear cell renal cell carcinoma ... physic-chemical properties (size and charge) of each separate material, including PCFC-g-PEI and FA-PEAs, as well as the FA-PEAs: pVHL complexes Because PCFC-g-PEI and FA-PEAs are amphiphilic ... was larger than the FA-PEAs:pVHL complexes obviously at same ratio Generally, the particle size of FA-PEAs:pVHL complexes was decreased along with the increase of the weight ratios between FA-PEAs...
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Báo cáo sinh học: "Safety and feasibility of third-party multipotent adult progenitor cells for immunomodulation therapy after liver transplantation–a phase I study (MISOT-I)" doc
... of interdisciplinary transplant medicine has made liver transplantation a standard-of-care clinical therapy for end-stage liver disease Long-term side effects of organ transplantation with chronic ... contrast, pulmonary function tests, and arterial blood gas analysis Intraoperative data (warm and cold ischemia Popp et al Journal of Translational Medicine 2011, 9:124 http://www.translational-medicine.com/content/9/1/124 ... lymphoproliferative disease and hepatocellular carcinoma) except for squamous or basal cell carcinoma of the skin that has been treated with no evidence of recurrence Unstable myocardium (evolving myocardial...
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