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Transcript
Gene Therapy Antisense Technologies antisense oligonucleotides(ODNs) Ribozymes DNAzymes RNA interference(RNAi). Gene Therapy... ----idea is to replace defective genes Gene therapy is a technique for correcting defective genes responsible for disease development. Several approaches for correcting faulty genes 1. A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. 2. An abnormal gene could be swapped for a normal gene through homologous recombination. 3. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. 4. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered. Deliver the therapeutic gene Some of the different types of viruses used as gene therapy vectors: Retroviruses Adenoviruses Adeno-associated viruses Herpes simplex viruses Deliver the therapeutic gene Adenoviruses Challenge Efficient delivery Enhanced stability Minimization of off-target effects Identification of sensitive sites in the target RNAs Status of gene therapy The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. Gene Therapy in the clinic Trials Wordldwide Phase I Phase I-II Phase II Phase II-III Phase III 66% Phase I 21% Phase I-II 11% Phase II 0.8% Phase II-III 0.7% Phase III in spite of 13 year- research only less than 1% of the trials has reached phase III For more details Genetic Modification Clinical Research Information System (GeMCRIS) http://www.gemcris.od.nih.gov/ Thanks~!! ~*_*~
