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	Presentation on Gene Therapy 3/26/03 Somatic Gene Therapy Karin Basic Definition of "Somatic Gene Therapy": The introduction of a fully functional gene into a target cell, with the aim of correcting a specific genetic disease. Monogenic Therapies Targets disorders caused by the mutation in a single gene Cystic fibrosis Sickle cell anemia Hemophilia A Aim Cure disease by introducing fully functional gene into the tissues of affected organs Multifactorial Gene Therapy Involves interaction of multiple genes, including possible involvement of environment and infectious agents Aim Induce or improve body’s own ability to heal or alter itself Covers broad array of therapies: HIV Cancer Treatments changing height and weight Current Performance of Gene Therapy Monogenic disorders Modified Viral Vectors Retroviruses Adenoviral Vectors Herpes Simplex Virus vector Ribozymes and Oligonucleotides – modify protein expression DNA/ Lipid complexes Problems Maloney based retrovirus only transduce dividing cells Lenti-virus Long-term expression of gene product is limited Limited number of patients Ribozymes and Oligonucleotides – modify protein expression DNA/ Lipid complexes Problems Maloney based retrovirus only transduce dividing cells Lenti-virus Long-term expression of gene product is limited Limited number of patients Multifactorial therapy Induce immune system to kill cancerous cells Addition of genes for TNF Induce suicide genes Addition of MDR genes- chemo Protective vaccines References: Textbook: Genetic Engineering, Science and Ethics on the new frontier http://www.fda.gov/fdac/features/2000/gene.html http://www.ornl.gov/TechResources/Human_Genome/medicine/genetherapy.html http://www.colby.edu/~wjgallit/st112project/Gene%20Therapy    ..
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