1. Gene Therapy

2. antisense oligonucleotides(ODNs) antisense oligonucleotides(ODNs) Ribozymes Ribozymes DNAzymes DNAzymes RNA interference(RNAi). RNA interference(RNAi). Antisense Technologies

3. Gene Therapy... ----idea is to replace defective genes ----idea is to replace defective genes Gene therapy is a technique for correcting defective genes responsible for disease development.

4. 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.

5. Deliver the therapeutic gene Some of the different types of viruses used as gene therapy vectors: Some of the different types of viruses used as gene therapy vectors: Retroviruses Retroviruses Adenoviruses Adenoviruses Adeno-associated viruses Adeno-associated viruses Herpes simplex viruses Herpes simplex viruses

6. Deliver the therapeutic gene Adenoviruses

7. Efficient delivery Efficient delivery Enhanced stability Enhanced stability Minimization of off-target effects Minimization of off-target effects Identification of sensitive sites in the target RNAs Identification of sensitive sites in the target RNAs Challenge

8. The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. Status of gene therapy

9. Gene Therapy in the clinic Trials Wordldwide 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

10. Genetic Modification Clinical Research Information System (GeMCRIS) Genetic Modification Clinical Research Information System (GeMCRIS) http://www.gemcris.od.nih.gov/ http://www.gemcris.od.nih.gov/http://www.gemcris.od.nih.gov/ For more details

11. Thanks~!! ~*_*~