000			05691cam a2200613Ii 4500
001			ocn895772596
003			OCoLC
005			20190328114809.0
006			m o d
007			cr cnu|||unuuu
008			141118s2015 ne a ob 001 0 eng d
040			_aN$T _beng _erda _epn _cN$T _dEBLCP _dN$T _dZCU _dOPELS _dOCLCF _dMYG _dOCLCQ _dUKMGB _dYDXCP _dYDX _dMERER _dOCLCO _dOCLCQ _dU3W _dD6H _dCHVBK _dAU@ _dWYU _dCUY _dLOA _dOCLCQ _dUSU _dMERUC _dOCLCO _dICG _dOCLCO _dK6U _dCOCUF _dVT2 _dDKC
016	7		_a016965687 _2Uk
016	7		_a016963762 _2Uk
019			_a897881152 _a898418256 _a907378542 _a992488055 _a1066623932 _a1088968408
020			_a9780128005644 _q(electronic bk.)
020			_a0128005645 _q(electronic bk.)
020			_z9780128005637
020			_z0128005637
035			_a(OCoLC)895772596 _z(OCoLC)897881152 _z(OCoLC)898418256 _z(OCoLC)907378542 _z(OCoLC)992488055 _z(OCoLC)1066623932 _z(OCoLC)1088968408
050		4	_aRB155.8
060		4	_a2015 A-003
060		4	_aQU 560
072		7	_aMED _x071000 _2bisacsh
082	0	4	_a615.8/95 _223
245	0	0	_aTranslating gene therapy to the clinic : techniques and approaches / _h[electronic resource] _cedited by Jeffrey Laurence, Michael Franklin.
264		1	_aAmsterdam : _bAcademic Press, an imprint of Elsevier, _c2015
264		4	_c�2015
300			_a1 online resource : _bcolor illustrations.
336			_atext _btxt _2rdacontent
337			_acomputer _bc _2rdamedia
338			_aonline resource _bcr _2rdacarrier
490	1		_aAdvances in translational research
588	0		_aOnline resource; title from PDF title page (Ebsco, viewed Dec. 18, 2014).
504			_aIncludes bibliographical references and index.
520			_aTranslating 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, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme.
505	0		_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.
505	8		_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.
505	8		_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.
505	8		_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.
650		0	_aGene therapy.
650		7	_aMEDICAL _xPharmacology. _2bisacsh
650		7	_aGene therapy. _2fast _0(OCoLC)fst00939638
650	1	2	_aGenetic Therapy _xmethods. _0(DNLM)D015316Q000379
650	2	2	_aTranslational Medical Research. _0(DNLM)D057170
655		0	_aElectronic book.
655		4	_aElectronic books.
655		7	_aElectronic books. _2lcgft
700	1		_aLaurence, Jeffrey, _eeditor.
700	1		_aFranklin, Michael, _eeditor.
776	0	8	_iPrint version: _tTranslating gene therapy to the clinic. _d[S.l.] : Elsevier Academic Press, 2015 _z0128005637 _z9780128005637 _w(OCoLC)884300585
830		0	_aAdvances in translational research.
856	4	0	_3ScienceDirect _uhttp://www.sciencedirect.com/science/book/9780128005637
999			_c247003 _d247003