LEADER 04307nam 2200613 a 450 001 9910780712703321 005 20230725041549.0 010 $a1-283-14380-1 010 $a9786613143808 010 $a1-61344-779-5 010 $a981-4280-91-7 035 $a(CKB)2490000000001950 035 $a(EBL)731223 035 $a(OCoLC)714877558 035 $a(SSID)ssj0000523036 035 $a(PQKBManifestationID)12233634 035 $a(PQKBTitleCode)TC0000523036 035 $a(PQKBWorkID)10557436 035 $a(PQKB)10824760 035 $a(MiAaPQ)EBC731223 035 $a(WSP)00007406 035 $a(Au-PeEL)EBL731223 035 $a(CaPaEBR)ebr10480253 035 $a(CaONFJC)MIL314380 035 $a(EXLCZ)992490000000001950 100 $a20110713d2010 uy 0 101 0 $aeng 135 $aur|n|---||||| 181 $ctxt 182 $cc 183 $acr 200 02$aA guide to human gene therapy$b[electronic resource] /$feditors, Roland W. Herzog, Sergei Zolotukhin 210 $aSingapore ;$aHackensack, N.J. $cWorld Scientific Pub. Co.$d2010 215 $a1 online resource (416 p.) 300 $aDescription based upon print version of record. 311 $a981-4280-90-9 320 $aIncludes bibliographical references and index. 327 $aPreface; Contents; Contributors; 1. Non-Viral Gene Therapy Sean M. Sullivan; 2. Adenoviral Vectors Stuart A. Nicklin and Andrew H. Baker; 3. Retroviral Vectors and Integration Analysis Cynthia C. Bartholomae, Romy Kirsten, Hanno Glimm, Manfred Schmidt and Christof von Kalle; 4. Lentiviral Vectors Janka Ma?trai, Marinee K. L. Chuah and Thierry VandenDriessche; 5. Herpes Simplex Virus Vectors William F. Goins, David M. Krisky, James B. Wechuck, Darren Wolfe, Justus B. Cohen and Joseph C. Glorioso; 6. Adeno-Associated Viral (AAV) Vectors Nicholas Muzyczka 327 $a7. Regulatory RNA in Gene Therapy Alfred. S. Lewin8. DNA Integrating Vectors (Transposon, Integrase) Lauren E. Woodard and Michele P. Calos; 9. Homologous Recombination and Targeted Gene Modification for Gene Therapy Matthew Porteus; 10. Gene Switches for Pre-Clinical Studies in Gene Therapy Caroline Le Guiner, Knut Stieger, Alice Toromanoff, Fabienne Rolling, Philippe Moullier and Oumeya Adjali; 11. Gene Therapy for Central Nervous System Disorders Deborah Young and Patricia A. Lawlor; 12. Gene Therapy of Hemoglobinopathies Angela E. Rivers and Arun Srivastava 327 $a13. Gene Therapy for Primary Immunodeficiencies Aisha Sauer, Barbara Cassani and Alessandro Aiuti14. Gene Therapy for Hemophilia David Markusic, Babak Moghimi and Roland Herzog; 15. Gene Therapy for Obesity and Diabetes Sergei Zolotukhin and Clive H. Wasserfall; 16. Gene Therapy for Duchenne Muscular Dystrophy Takashi Okada and Shin'ichi Takeda; 17. Cancer Gene Therapy Kirsten A.K. Weigel-Van Aken; 18. Gene Therapy for Autoimmune Disorders Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins; 19. Gene Therapy for Inherited Metabolic Storage Diseases Cathryn Mah 327 $a20. Retinal Diseases Shannon E. Boye, Sanford L. Boye and William W. Hauswirth21. A Brief Guide to Gene Therapy Treatments for Pulmonary Diseases Ashley T. Martino, Christian Mueller and Terence R. Flotte; 22. Cardiovascular Disease Darin J. Falk, Cathryn S. Mah and Barry J. Byrne; Index 330 $aEver since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy - though still in its infancy - remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ( vector) to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in nume 606 $aGene therapy 615 0$aGene therapy. 676 $a615.895 701 $aZolotukhin$b Sergei$01583507 701 $aHerzog$b Roland W$01179740 801 0$bMiAaPQ 801 1$bMiAaPQ 801 2$bMiAaPQ 906 $aBOOK 912 $a9910780712703321 996 $aA guide to human gene therapy$93866653 997 $aUNINA