04304nam 2200625 a 450 991080911020332120240516083415.01-283-14380-197866131438081-61344-779-5981-4280-91-7(CKB)2490000000001950(EBL)731223(OCoLC)714877558(SSID)ssj0000523036(PQKBManifestationID)12233634(PQKBTitleCode)TC0000523036(PQKBWorkID)10557436(PQKB)10824760(MiAaPQ)EBC731223(WSP)00007406(Au-PeEL)EBL731223(CaPaEBR)ebr10480253(CaONFJC)MIL314380(EXLCZ)99249000000000195020110713d2010 uy 0engur|n|---|||||txtccrA guide to human gene therapy /editors, Roland W. Herzog, Sergei Zolotukhin1st ed.Singapore ;Hackensack, N.J. World Scientific Pub. Co.20101 online resource (416 p.)Description based upon print version of record.981-4280-90-9 Includes bibliographical references and index.Preface; 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 Mátrai, 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 Muzyczka7. 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 Srivastava13. 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 Mah20. 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; IndexEver 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 numeGene therapyGene therapy.615.895Zolotukhin Sergei1620937Herzog Roland W1179740MiAaPQMiAaPQMiAaPQBOOK9910809110203321A guide to human gene therapy3954011UNINA