novel gene therapy approaches

... Introduction Gene therapy is a promising approach to treat intractable and refractory diseases at the genetic level Basically, in gene therapy, target gene expression is induced by delivering foreign genes ... mice Cancer Gene Therapy 2012;19(2) 77-83 17 18 Novel Gene Therapy Approaches [16] Liu T, Zhang G, Chen YH, Chen Y, Liu X, Peng J, Xu MH, Yuan JW Tissue specific expression of suicide genes delivered ... of target gene expression or gene silencing can also be performed using miRNA, siRNA or shRNA expression vectors [1] Gene therapy is useful for both genetic and acquired diseases For genetic diseases,...

Ngày tải lên: 23/03/2014, 14:20

... excision, radiotherapy and chemotherapy Currently, gene therapy has emerged as a promising new approach for treating aggressive malignant tumors; however, the low efficiency of transgene delivery ... expression of transgenes The vector has a high cloning capacity, allowing for the accommodation and delivery of large functional genes or multiple genes Unlike many other gene therapy viral vectors, ... tumor-targeted gene therapy that improves the treatment of patients with breast cancer and overcomes the hurdles faced by stem cell-based gene therapy via two main objectives: 1) To develop therapeutic genes...

Ngày tải lên: 09/09/2015, 10:06

... 1.3.3 Gene therapy strategies for bladder cancer 12 1.3.3.1 Immune inductive gene therapy 12 1.3.3.2 Corrective gene therapy 13 1.3.3.3 Cytotoxic gene therapy 14 1.3.3.4 Anti-sense oncogene therapy ... 1.4 Cytokine gene therapy of cancer 17 1.4.1 Introduction 17 1.4.2 Modality of gene transfer for cytokine gene therapy 17 1.4.3 The target cells of gene transfer for cytokine gene therapy 18 1.4.4 ... mdr-1 c-erb-2 Ras Tumor Suppressor gene Tumor Suppressor gene Tumor Suppressor gene Tumor Suppressor gene Oncogene Oncogene Oncogene Oncogene Inactivation Inactivation Inactivation Inactivation...

Ngày tải lên: 08/11/2015, 16:45

... and E2b genes and the use of homologous (rat) BMP genes not improve the osteogenic potential of direct BMP adenovirus vector gene therapy, the results of this study indicate that BMP gene therapy ... for generating recombinant adenoviruses Proc Natl Acad Sci U S A 1998; 95:2509-14 19 Helm GA, Alden TD, Sheehan JP, Kallmes D Bone morphogenetic proteins and bone morphogenetic protein gene therapy ... the vector and to its transgene J Immunol 1997; 159:685-93 43 Varady P, Li JZ, Cunningham M, et al Morphologic analysis of BMP-9 gene therapy- induced osteogenesis Hum Gene Ther 2001; 12:697-710...

Ngày tải lên: 31/10/2012, 17:08

... related to human germline gene therapy Criteria for Assessing Applicability to Human Germline Gene Therapy An ideal gene transfer system in the context of human germline gene therapy would have the ... for human germline gene therapy The lack of human ESCs leaves NT-based gene transfer as the only method that might be able to permit gene targeting in human germline gene therapy NT could probably ... Chromosome engineering: prospects for gene therapy Gene Ther 2002; 9(11):713-718 85 Lipps HJ, Jenke ACW, Nehlsen K, et al Chromosome-based vectors for gene therapy Gene Ther 2003; 304:23-33 86 Abbondanzo...

Ngày tải lên: 03/11/2012, 10:01

... tạo số lượng lớn, chuyển vào người bệnh nguyenthanhloi_2@yahoo.com 13 Kỹ thuật dùng súng bắn gen (gene gun): Kỹ thuật bắn gen nhờ súng bắn gen để chuyển gen liệu pháp vào tế bào đích thường đạt ... + Kích thích gen thể để tăng cường miễn dịch chống phát triển HIV + Sử dụng gen tự sát (Suicide gene) biến đổi làm cho tế bào nhiễm HIV tự chết + Tạo vacine phòng chống nhiễm HIV công nghệ di ... dụng gen đột biến Rev10, gen tăng cường hoạt động gen cytokin, sử dụng gen tự tử gen độc tố (toxic gene) Cũng sử dụng liệu pháp gen tăng cường hoạt động gen cytokin tổng hợp protein đáp ứng miễn...

Ngày tải lên: 06/09/2013, 22:49

... unknown genes We have previously revealed that several of them are novel genes, such as factor for adipocyte differentiation (fad) 24, fad104 and fad158, which play crucial roles in adipogenesis ... seems to play a role in adipogenesis To characterize the function of this gene during adipogenesis, we performed RNAi to silence the expression of fad49 during adipogenesis For the RNAi experiments, ... [16,17] About half of them were unknown genes As a result of attempts to isolate fulllength cDNAs for the unknown genes, we previously isolated several novel genes that are crucial for adipocyte...

Ngày tải lên: 16/03/2014, 04:20

... for Gene Therapy 109 Aurore Burgain-Chain and Daniel Scherman Chapter siRNA and Gene Formulation for Efficient Gene Therapy 135 Ian S Blagbrough and Abdelkader A Metwally VI Contents Section Gene ... addressed and the gene types transferred in gene therapy clinical trials, re spectively [6] Indications addressed by gene therapy clinical trials Cancer diseases Cardiovascular diseases Gene marking ... effi cient and safe gene therapy While the length of time a gene must be expressed for efficacy depends on both the therapeutic strategy and the disease, many gene therapy approaches prove ineffective...

Ngày tải lên: 17/03/2014, 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 ... 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

... vitro Gene Ther 4, 63–75 Pan, D., Aronovich, E.L., McIvor, R.S & Whitley, C.B (2000) Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I Gene ... vector to mediate gene transfer and expression in appropriate targets in vivo (muscle, brain, hematopoietic cells) and correct GAG storage in murine MPS I cells as a model for gene therapy of human ... particularly important in the gene transfer approaches to treatment of mucopolysaccharidosis type I because they imply that the introduction of a therapeutic IDUA gene by lentiviral vector into...

Ngày tải lên: 24/03/2014, 04:21

... 130 IAP Gene Therapy for Stroke 131 IAP Gene Therapy for Optic Neurodegeneration 132 IAP Gene Therapy for Parkinson’s Disease 133 Prospects for IAP-based Gene Therapy ... 130 IAP Gene Therapy for Stroke 131 IAP Gene Therapy for Optic Neurodegeneration 132 IAP Gene Therapy for Parkinson’s Disease 133 Prospects for IAP-based Gene Therapy ... Adenovirus-Mediated Inducible Gene Transduction 198 Future Applications 198 MEDICAL INTELLIGENCE UNIT 15 Adenoviruses: Basic Biology to Gene Therapy Prem Seth, Ph.D Human Gene Therapy Research...

Ngày tải lên: 10/04/2014, 10:58

... Human Gene Therapy Further government hearings and investigations of the gene therapy research field revealed that a total of 731 adverse events had occurred in human studies involving gene therapy ... regulators reflect on first gene therapy death Nat Med 6:6, 2000 Jenks S Gene therapy death—“everyone has to share in the guilt.” J Natl Cancer Inst 92:98–100, 2000 Marchall E Gene therapy death prompts ... community acceptance in principle of the appropriateness of gene therapy for somatic cells in humans for specific diseases Somatic gene therapy was and is seen as an extension of current experimental...

Ngày tải lên: 10/04/2014, 22:10

... utility REQUIREMENTS FOR GENE TRANSFER INTO HEMATOPOIETIC CELLS Ex Vivo Versus in Vivo Gene Transfer Specific aspects of gene transfer techniques are advantageous for gene therapy approaches when applied ... 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 ... revolutionize the gene therapy field Immune Responses to Vectors and Transgenes Immune responses against vector proteins or transgene-encoded proteins are clearly an obstacle to successful gene therapy...

Ngày tải lên: 10/04/2014, 22:10

... in the case of gene therapy as well Appropriate Candidates for Gene Therapy We now turn to another dimension of ethics issues in gene therapy, namely, who the candidates are for therapy We distinguish ... the case of Donald and the gene therapy clinical trial we are considering for his CF? 326 ETHICAL ISSUES IN MOLECULAR MEDICINE AND GENE THERAPY In the case of gene therapy there appear to be ... regulate gene therapy? The point of that question may be a little obscure at first There has been no public uproar regarding gene therapy Do we have any reason for believing that gene therapy should...

Ngày tải lên: 10/04/2014, 22:10

... and medicine GENE THERAPY: CURRENT BASIC SCIENCE ISSUES Two critical steps are required for gene therapy using gene transfer techniques: (1) the appropriate transfer of gene( s) or genetic material ... therapy Gene Targeting Gene therapy approaches could be enhanced by directing gene transfer and expression to specific cells or tissues (see Chapter 5) The easiest approach would be ex HUMAN GENE THERAPY: ... studies are gene- marking studies where a cell is marked with a gene to elucidate metatasis or recurrence GENE THERAPY: CURRENT BASIC SCIENCE ISSUES 15 Adverse short-term effects of gene therapy...

Ngày tải lên: 10/04/2014, 22:10

... 78 VECTORS OF GENE THERAPY VIRAL VECTORS USED FOR GENE THERAPY Based on the virus life cycle, infectious virions are very efficient at transferring genetic information Most gene therapy experiments ... the relatively low levels of gene expression achieved in animals are problematic For currently selected genes used for gene therapy, the level of expression of the gene product does not need to ... use of immunosuppressive therapy could provide persistent gene expression following adenovirus-mediated gene transfer and allow secondary gene transfer A variety of approaches to suppress the...

Ngày tải lên: 10/04/2014, 22:11

... 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 ... for gene therapy approaches to prevent liver fibrosis due to ethanol consumption 178 GENE THERAPY FOR LIVER DISEASE FIGURE 7.10 Possible biological mechanisms, as proposed by Thurman, for the generation ... chronic disease of the liver and are serious threats to public health Gene therapy approaches to therapy are the 180 GENE THERAPY FOR LIVER DISEASE use of targeted antisense molecules, ribozymes...

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 ... 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 in man: ... transfection For gene therapy of the heart, genetic manipulation of the myocardium has generated considerable interest in the use of gene transfer of the b-adrenergic recepter gene into the ailing...

Ngày tải lên: 10/04/2014, 22:11

... treatment by gene therapy approaches to inhibit the replication of HIV-1 The ultimate goal of gene therapy is to inhibit HIV-1 viral replication and the resulting AIDS pathogenesis For 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 ... that they become dysfunctional GENETIC APPROACHES TO INHIBIT HIV REPLICATION Approaches to gene therapy for HIV can be divided into three broad categories: (i) protein approaches such as transdominant...

Ngày tải lên: 10/04/2014, 22:11

... normal gene or by directly correcting the gene defect If a gene incurs a gain in function, such as for an oncogene or growth factor, then approaches at gene deletion or regulation of gene expression ... Suppressor Genes Tumor suppressor genes are a genetically distinct class of genes involved in suppressing abnormal growth Loss of function of tumor suppressor proteins results in GENE THERAPY APPROACHES ... best targets of antisense gene therapy in cancer are overexpressed oncogenes that play a role in pathogenesis Numerous oncogenes have been targeted for antisense gene therapy They include c-fos...

Ngày tải lên: 10/04/2014, 22:11