monitoring duchenne muscular dystrophy gene therapy with epitope specific monoclonal antibodies

... together with the fact that the tumor specific hTERT promoter was sufficient to drive suicide gene expression indicated that adenovirus-mediated HRP gene therapy combined with cytokine IL-12 gene therapy ... therapeutic gene to cancer cells Gene directed enzyme/prodrug therapy (GDEPT) or suicide gene therapy using viral vectors is an attractive alternative approach to cancer therapy, with the potential ... therapeutic efficacy of suicide gene therapy, it is important to explore new strategies of combined therapy, which employ targeted suicide gene in combination with immunotherapy that cooperatively...

Ngày tải lên: 18/06/2014, 19:20

... together with the fact that the tumor specific hTERT promoter was sufficient to drive suicide gene expression indicated that adenovirus-mediated HRP gene therapy combined with cytokine IL-12 gene therapy ... therapeutic gene to cancer cells Gene directed enzyme/prodrug therapy (GDEPT) or suicide gene therapy using viral vectors is an attractive alternative approach to cancer therapy, with the potential ... therapeutic efficacy of suicide gene therapy, it is important to explore new strategies of combined therapy, which employ targeted suicide gene in combination with immunotherapy that cooperatively...

Ngày tải lên: 20/06/2014, 03:20

... Nair et al.: Successful bone marrow transplantation in a patient with Diamond-Blackfan anemia with co-existing Duchenne muscular dystrophy: a case report Journal of Medical Case Reports 2011 5:216 ... van Ommen GJ: Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications Am J Hum Genet 1989, 45:835-847 Gussoni E, ... persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation J Clin Invest 2002, 110:807-814 Cooke R: Stem cells aimed at muscular dystrophy: evidence suggests...

Ngày tải lên: 10/08/2014, 23:21

... the dogs of the gene therapy group (n = 4) through intrathecal injection using a 27-gauge needle Before this administration, the dogs of the gene therapy group were anesthetized with zoletil Pyridoxine ... showed that the gene cloned in pcDNA1 had a high degree of sequence homology with other mammalian β-NGF genes The pcDNA1 sequence shared 86% and 83% sequence homology with that of human (GeneBank sequence ... cytoplasm (arrows) in a few neurons in the gene therapy group H&E stain, ×200 disrupted with vacuolation in the positive control group (Fig 3B) In the gene therapy group, swollen axons were occasionally...

Ngày tải lên: 07/08/2014, 23:22

... for gene therapy Gene Ther 2004, 11:1321-1330 37 Weeratna RD, Wu T, Efler SM, Zhang L, Davis HL: Designing gene therapy vectors: avoiding immune responses by using tissue -specific promoters Gene ... sequence of the tetO and influence gene regulation with the tetracycline system Mol Ther 2004, 10:127-138 31 Bestor TH: Gene silencing as a threat to the success of gene therapy J Clin Invest 2000, ... to determine the inhibitor with the ideal pharmacokinetic profile for gene therapy application Conclusion Anti-TNF treatment for RA could be delivered safely by gene therapy through the use of...

Ngày tải lên: 09/08/2014, 10:20

... Amplification” CHAPTER INTRODUCTION 1.1 Duchenne muscular dystrophy 1.1.1 Characteristics of DMD Duchenne muscular dystrophy (DMD) was first described by Duchenne de Boulogne, the French neurologist, ... daughters will carry the gene A carrier mother may or may not pass on the gene with the mutation (figure 2) Figure 2: Genetic of DMD Resource: http://duchennemusculardystrophy-2.wikispaces.com/ ... muscle wasting which characterizes Duchenne muscular dystrophy 1.4 Mutations in the dystrophin gene Mutations in the large dystrophin gene, which consists of 79 exons generally causes a disruption...

Ngày tải lên: 05/11/2015, 17:38

... detection in families affected by Duchenne muscular dystrophy using Multiplex Ligation- dependent Probe Amplification” CHAPTER INTRODUCTION 1.1 Duchenne muscular dystrophy 1.1.1 Characteristics ... lặp đoạn exon 11-20 51-60 gen dystrophy Kỹ thuật MLPA thực theo khuyến nghị nhà sản xuất PREFACE Duchenne muscular dystrophy (DMD) is one of the most common fatal genetic disorders affecting children ... TÓM TẮT Bệnh loạn dưỡng Duchenne (Duchenne Muscular Dystrophy- DMD) bệnh di truyền lặn liên kết với nhiễm sắc thể X gây đột biến gen dystrophin.Theo...

Ngày tải lên: 05/11/2015, 17:39

... Hege KM: A phase II randomized study of HIV -specific T-cell gene therapy in subjects with undetectable plasma viremia on combination antiretroviral therapy Mol Ther 2002, 5:788-797 Walker RE, ... http://www.retrovirology.com/content/1/1/29 Yu M, Poeschla E, Wong-Staal F: Progress towards gene therapy for HIV infection Gene Ther 1994, 1:13-26 Bridges SH, Sarver N: Gene therapy and immune restoration for HIV disease Lancet 1995, ... antiHIV therapy will require the construction of high-titer vectors, with the aim of increasing the proportion of vectortransduced HIV target cells An alternative method for IFN-β gene therapy...

Ngày tải lên: 13/08/2014, 13:20

... Genetic Vaccines and Therapy 2008, 6:8 Background Gene therapy represents a potential new modality for the treatment of cancer and it is developing with a very fast pace ... studied and most widely used system in cancer gene therapy They are of particular utility for cancer gene therapy applications, where temporary gene expression is acceptable or even beneficial ... the HSV TK gene to patients with mesothelioma via intrapleural administration have been reported [43] Gene Page of 12 (page number not for citation purposes) Genetic Vaccines and Therapy 2008,...

Ngày tải lên: 14/08/2014, 19:22

... 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 ... implications for gene therapy Gene Ther 1997; 4:258-63 Amalfitano A, Hauser MA, Hu H, et al Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted ... 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...

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 ... of many human genes Additionally, control possibilities are less with RVV delivered transgenes compared with transgenes delivered by microinjection The safety problems associated with RVVs (insertional ... 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...

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

... of muscular dystrophy The other major types of muscular dystrophy include: • • • • • Limb-girdle muscular dystrophy Congenital muscular dystrophy Oculopharyngeal muscular dystrophy Distal muscular ... muscular dystrophy Distal muscular dystrophy Emery-Dreifuss muscular dystrophy Limb-girdle muscular dystrophy Muscles usually affected first by this form of muscular dystrophy include: • • Hips Shoulders ... progresses slowly Causes Muscular dystrophy is a general term for a group of inherited diseases involving a defective gene Each form of muscular dystrophy is caused by a genetic mutation that's...

Ngày tải lên: 19/02/2014, 19:20

... oculopharyngeal muscular dystrophy Neuromusc Disord 7, 63–69 ´ 16 Calado A, Tome FMS, Brais B, Rouleau GA, Kuhn U, ¨ Wahle E & Carmo-Fonseca M (2000) Nuclear inclusions in oculopharyngeal muscular dystrophy ... oculopharyngeal muscular dystrophy Hum Mol Genet 15, 23–31 25 Uyama E, Tsukahara T, Goto K, Kurano Y, Ogawa M, Kim Y-J, Uchino M & Arahata K (2000) Nuclear accumulation of expanded PABP2 gene product ... in fermentors of L culture volumes with 5% yeast extract (Roth, Karlsruhe, Germany) Feeding with yeast extract was started at D600 ¼ 10 Cells were induced with mm isopropyl thiob-d-galactoside...

Ngày tải lên: 07/03/2014, 11:20

... is encoded by the EMD gene on chromosome Xq28 and mutations within this gene give rise to X-linked Emery–Dreifuss muscular dystrophy (X-EDMD), a neuromuscular condition with an associated life-threatening ... nuclear membrane in 14 patients with Emery–Dreifuss muscular dystrophy Nat Genet 12, 254–259 Manilal S, thi Man N, Sewry CA & Morris GE (1996) The Emery–Dreifuss muscular dystrophy protein, emerin, ... species cross-reacted with an antiphosphotyrosine antibody but, perhaps not surprisingly, the commercial antiphosphoserine and threonine antibodies used did not cross-react with any of the emerin...

Ngày tải lên: 17/03/2014, 17:20

... AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy 429 Takashi Okada Applications: Inhereted Diseases 451 Chapter 18 Gene Therapy for Primary Immunodeficiencies ... 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...

Ngày tải lên: 17/03/2014, 21:20

... X-linked gene responsible for Emery–Dreifuss muscular dystrophy Nat Genet 8, 323–327 Emery, A.E.H (1989) Emery–Dreifuss syndrome J Med Genet 26, 637–641 Emery, A.E.H (2000) Emery–Dreifuss muscular dystrophy ... Emery–Dreifuss muscular dystrophy phenotype J Cell Sci 111, 781–792 22 Morris, G.E & Manilal, S (1999) Heart to heart: from nuclear proteins to Emery–Dreifuss muscular dystrophy Hum Mol Genet 8, 1847–1851 ... T & Arahata, K (1996) Emerin deficiency at the nuclear membrane in patients with Emery–Dreifuss muscular dystrophy Nat Genet 12, 254–259 20 Yorifuji, H., Tadano, Y., Tsuchiya, Y., Ogawa, M., Goto,...

Ngày tải lên: 23/03/2014, 12:20

... 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 ... 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, ... CY Tumor-targeted gene therapy using Adv-AFP-HRPC/IAA prodrug system suppresses growth of hepatoma xeno‐ grafted in mice Cancer Gene Therapy 2012;19(2) 77-83 17 18 Novel Gene Therapy Approaches...

Ngày tải lên: 23/03/2014, 14:20

... incubation fibroblasts grown on coverslips were fixed with 3.7% formaldehyde in NaCl/Pi, washed once with 0.1 M glycine in NaCl/Pi, twice with NaCl/Pi and once with H2O The fluorescence was visualized under ... 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 ... lentivirus-mediated gene transfer Hum Gene Ther 11, 1139–1150 Dull, T., Zufferey, R., Kelly, M., Mandel, R.J., Nguyen, M., Trono, D & Naldini, L (1998) A third-generation lentivirus vector with a conditional...

Ngày tải lên: 24/03/2014, 04:21

... and 5¢ halves of the TDH genes The remaining fragments containing the 3¢ halves of the TDH genes were ligated with fragments from the plasmid ASAJ2682 containing the kan gene as a selective marker ... blotting using antibodies to Gts1p (upper panel) The cell lysates were immunoprecipitated with antibodies to GAPDH, and coprecipitated Gts1p was detected by Western blotting using antibodies to ... oligonucleotide 15 (Table 1), which is identical with the complementary sequence between + 371 and + 435, with respect to the first nucleotide A of each gene, having 30% dissimilarity from the corresponding...

Ngày tải lên: 31/03/2014, 21:21