molecular medicine and gene therapy an introduction

... Ser-Asp-His [10,12]. To obtain an insight into the physiological and cellular function of the putative lysosomal SC Scpep1, we analysed the molecular properties of Scpep1 and generated an Scpep1 gene trap (Scpep1-gt) ... kDa Scpep1 in cell extracts and in the medium of HT1080- Scpep1 cells (Fig. 1A, lanes 3 and 4), but not in untransfected HT1080 cells (lanes 1 and 2). Unexpectedly, an additional 18 kDa form of ... activity (A) and analysed by western blot analyses (B), using the Scpep1 antiserum to detect the 35 kDa fragment and an anti- Ctsa rat IgG2B to detect the 32 kDa heavy chain of Ctsa. K. Kollmann et...

Ngày tải lên: 07/03/2014, 03:20

... CHAPTER Molecular Medicine and Gene Therapy: An Introduction THOMAS F KRESINA, PH.D and ANDREA D BRANCH, PH.D INTRODUCTION The use of genetics and genetic manipulation by humans for the therapy ... of some genes is up-regulated and of other genes down-regulated (Adapted from Anderson and Anderson, Electrophoresis, 1996.) MOLECULAR MEDICINE AND GENE THERAPY: AN INTRODUCTION gene and the ... fetal gene therapy Semin Perinatal 23:524–534, 1999 Zanjani ED, Anderson WF Prospects for in utero human gene therapy Science 285:2084– 2088, 1999 Gene Therapy, Disease Pathogenesis, and Transplantation...

Ngày tải lên: 10/04/2014, 22:10

... Chimeric animals can be generated through xenotransplantation, the transfer of tissue from one species into another species Xenotransplantation broadens therange of experimental manipulations and ... every gene transfer technique devel-oped, including retroviral and adenoviral vector infection, direct gene transfer,receptor-mediated gene transfer, and surgical transfer of genetically manipu-lated ... MICE 69 Trang 3chemical and physiological functions in mice and humans A unique model to studyhuman pathology in animals as well as murine/human biochemistry and physiologyis the chimeric animal...

Ngày tải lên: 10/08/2014, 14:22

... minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells Hum Mol Genet 5:913–921, 1996 An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F Kresina, ... multiple myeloma and breast cancer transplanted with genetically marked bone marrow and peripheral blood cells However, the marrow and blood cells were CD34enriched before transduction, thus ... 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...

Ngày tải lên: 10/08/2014, 14:22

... 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 ... dominant, most HD patients carry one copy of the expanded triplet gene and one normal copy of the gene Therefore, each of their children has a 50/50 chance of receiving the gene and a 50/50 chance ... techniques and an improved understanding of basal ganglia CLINICAL NEURODEGENERATIVE CONDITIONS 225 function and organization has revitalized the surgical treatments for Parkinson’s Magnetic resonance...

Ngày tải lên: 10/08/2014, 14:22

... both viral and nonviral vectors Tumor targeting and specificity need to be advanced and a further understanding of gene regulation, apoptosis, and the synergy between gene therapy and chemotherapy ... Basics of cancer gene therapy Anticancer Res 17:2887–2890, 1997 Genetic Basis of Carcinogenesis Hauses M, Schackert HK Gene therapy and gastrointestinal cancer: Concepts and clinical facts Langenbecks ... Neurological Disorders and Brain Tumors Humana, Totowa, NJ, 1998 Doering LC Gene therapy and neurodegeneration Clin Neurosci 3:259–321, 1996 Kaplitt MG, Loewy AD Viral Vectors, Gene Therapy and Neuroscience...

Ngày tải lên: 10/08/2014, 14:22

... deficiency, hemophilia A and B) provide larger markets where gene therapy could have an impact As with any new therapy, gene therapy approach for a disease state would need to have advantages over treatments ... standards supported by validated analytical methods and controlled unit operations All components of the process must be generally recognized as safe and must meet all applicable regulatory standards ... support is the necessity of cleaning in place and sanitization by cycles of caustic washing The ability to withstand repeated cycles of regeneration, sterilization, and sanitization with 0.5 N NaOH...

Ngày tải lên: 10/08/2014, 14:22

... Issues in Molecular Medicine and Gene Therapy LEONARD M FLECK, PH.D BACKGROUND The presentation of ethical issues in molecular medicine and gene therapy warrants definition of terms An ethic, ... especially when that care can make the difference between life and death or 328 ETHICAL ISSUES IN MOLECULAR MEDICINE AND GENE THERAPY the difference between permanent injury and the healing of that ... of moral 336 ETHICAL ISSUES IN MOLECULAR MEDICINE AND GENE THERAPY guidance in standard clinical circumstances, for example, a child afflicted with lifethreatening cancer We saw that in our discussion...

Ngày tải lên: 10/04/2014, 22:10

... 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

... 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

... 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

... 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

... immunosuppressive agents and malignancy Curr Opin Organ Transplant 2004, 9:394 10 Vajdic CM, van Leeuwen MT: Cancer incidence and risk factors after solid organ transplantation Int J Cancer 2009, 125:1747-1754 ... 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 ... portal vein during transplantation and later intravenously on day analysis to screen for anti-donor antibodies and cytokines Moreover, we will analyze peripheral blood samples for genes that have...

Ngày tải lên: 18/06/2014, 22:20