LEADER 05384nam 2200649 450 001 9910787927203321 005 20200520144314.0 010 $a0-12-800564-5 035 $a(CKB)2670000000577155 035 $a(EBL)1849107 035 $a(SSID)ssj0001415944 035 $a(PQKBManifestationID)11836616 035 $a(PQKBTitleCode)TC0001415944 035 $a(PQKBWorkID)11352641 035 $a(PQKB)10917660 035 $a(Au-PeEL)EBL1849107 035 $a(CaPaEBR)ebr10991252 035 $a(CaONFJC)MIL663685 035 $a(OCoLC)895772596 035 $a(MiAaPQ)EBC1849107 035 $a(EXLCZ)992670000000577155 100 $a20141211h20152015 uy 0 101 0 $aeng 135 $aur|n|---||||| 181 $ctxt 182 $cc 183 $acr 200 00$aTranslating gene therapy to the clinic $etechniques and approaches /$fedited by Jeffrey Laurence, Michael Franklin 210 1$aLondon, England :$cAcademic Press,$d2015. 210 4$dİ2015 215 $a1 online resource (347 p.) 225 0 $aAdvances in Translational Research 300 $aDescription based upon print version of record. 311 $a1-322-32403-4 311 $a0-12-800563-7 320 $aIncludes bibliographical references at the end of each chapters and index. 327 $aFront Cover; Translating Gene Therapy to the Clinic: Techniques and Approaches; Copyright; Contents; Preface; REFERENCES; About the Editors; Contributors; Chapter 1 - Translating Genome Engineering to Survival; 1.1 ORIGINS; 1.2 SYNCHRONICITY OF DISCOVERIES; 1.3 GENE ADDITION; 1.4 FROM GENE ADDITION TO GENE EDITING; 1.5 THERAPY FOR GENETIC DISORDERS; 1.6 ROADMAP TO THE FUTURE; CONFLICT OF INTEREST; ACKNOWLEDGMENTS; REFERENCES; Chapter 2 - Pluripotent Stem Cells and Gene Therapy; 2.1 GENETIC APPROACHES TO PLURIPOTENCY; 2.2 TRANSCRIPTION FACTORS IMPORTANT FOR REPROGRAMMING TO PLURIPOTENCY 327 $a2.3 METHODS FOR GENETIC REPROGRAMMING2.4 CLINICAL TRANSLATION OF IPSCS; 2.5 CONCLUSION; REFERENCES; Chapter 3 - Genome Engineering for Therapeutic Applications; 3.1 INTRODUCTION; 3.2 CUSTOMIZABLE DNA-TARGETING PROTEINS; 3.3 GENOME EDITING WITH ENGINEERED NUCLEASES; 3.4 SYNTHETIC TRANSCRIPTION FACTORS FOR THERAPEUTIC APPLICATIONS; 3.5 CONCLUSION; ACKNOWLEDGMENTS; REFERENCES; Chapter 4 - Immune System Obstacles to In vivo Gene Transfer with Adeno-Associated Virus Vectors; 4.1 INTRODUCTION; 4.2 AAV VECTORS; 4.3 INNATE IMMUNITY IN AAV GENE TRANSFER; 4.4 T-CELL RESPONSES TO VECTORS 327 $a4.5 HUMORAL IMMUNITY4.6 CONCLUSIONS; ACKNOWLEDGMENT; REFERENCES; Chapter 5 - Risks of Insertional Mutagenesis by DNA Transposons in Cancer Gene Therapy; 5.1 INSERTIONAL MUTAGENESIS-THE DOWNSIDE OF GENE THERAPY?; 5.2 SLEEPING BEAUTY TRANSPOSON/TRANSPOSASE SYSTEM ADAPTED FOR GENE THERAPY; 5.3 PLASTICITY OF GENOMES AND GENE EXPRESSION IN HUMANS; 5.4 TRANSPOSON-MEDIATED GENE THERAPY IN THE CLINIC; 5.5 CONCLUSIONS; ACKNOWLEDGMENT; REFERENCES; Chapter 6 - Arthritis Gene Therapy: A Brief History and Perspective; 6.1 INTRODUCTION; 6.2 CONCEPTION AND STRATEGIES; 6.3 TECHNOLOGY DEVELOPMENT 327 $a6.4 UNRESOLVED ISSUES6.5 CLINICAL TRIALS; 6.6 VETERINARY APPLICATIONS; 6.7 OTHER APPLICATIONS OF INTRA-ARTICULAR GENE THERAPY; 6.8 COMMERCIALIZATION; 6.9 PERSPECTIVES; ACKNOWLEDGMENTS; REFERENCES; Chapter 7 - Type 1 Diabetes Mellitus: Immune Modulation as a Prerequisite for Successful Gene Therapy Strategies; 7.1 INTRODUCTION; 7.2 EFFECTIVE IMMUNE THERAPY/MODULATION: A PREREQUISITE FOR SUCCESSFUL GENE THERAPY OF TYPE 1 DIABETES; 7.3 TARGETED ISLET ANTIGEN RECOGNITION AND ANTIGEN-BASED THERAPIES; 7.4 BROAD IMMUNOSUPPRESSIVE THERAPIES; 7.5 IMMUNOTHERAPIES THAT TARGET EVENTS IN T CELL RESPONSE 327 $a7.6 PROSPECTS FOR IMMUNOTHERAPY IN PROTECTING NEO-BETA CELLS7.7 CONCLUSION; ACKNOWLEDGMENT; REFERENCES; Chapter 8 - Gene Therapy for Diabetes; LIST OF ABBREVIATIONS; 8.1 INTRODUCTION; 8.2 GENERATION OF ? CELLS FROM PANCREATIC MATURE NON-? CELLS; 8.3 GENERATION OF ? CELLS FROM TISSUE PROGENITOR CELLS; 8.4 GENERATION OF ? CELLS FROM STEM CELLS; 8.5 GENERATION OF NEW ? CELLS BY INDUCING THEIR REPLICATION; 8.6 CLOSING REMARKS; ACKNOWLEDGMENTS; REFERENCES; Chapter 9 - Gene Therapy for Neurological Diseases; 9.1 INTRODUCTION; 9.2 VIRAL VECTORS FOR NEUROLOGICAL DISEASES 327 $a9.3 GENE THERAPY FOR CHRONIC PAIN 330 $a Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, 606 $aGene therapy 606 $aHuman genetics 615 0$aGene therapy. 615 0$aHuman genetics. 676 $a616.042 702 $aLaurence$b Jeffrey 702 $aFranklin$b Michael 801 0$bMiAaPQ 801 1$bMiAaPQ 801 2$bMiAaPQ 906 $aBOOK 912 $a9910787927203321 996 $aTranslating gene therapy to the clinic$93786697 997 $aUNINA