1 / 11

Gene Therapy

Gene Therapy. Antisense Technologies. antisense oligonucleotides(ODNs) Ribozymes DNAzymes RNA interference(RNAi). Part of Gene Therapy. Gene Therapy... ----idea is to replace defective genes. Gene therapy is a technique for correcting defective genes responsible for disease development.

galeno
Download Presentation

Gene Therapy

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Gene Therapy

  2. Antisense Technologies • antisense oligonucleotides(ODNs) • Ribozymes • DNAzymes • RNA interference(RNAi). Part of Gene Therapy

  3. Gene Therapy... ----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 • A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. • An abnormal gene could be swapped for a normal gene through homologous recombination. • The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. • 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: • Retroviruses • Adenoviruses • Adeno-associated viruses • Herpes simplex viruses

  6. Deliver the therapeutic gene Adenoviruses

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

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

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

  11. Thanks~!! ~*_*~

More Related