1. Gene Therapy for the Treatment of Cystic Fibrosis Stephanie M. Delgado RISE Program BIOL 3095

2. Cystic Fibrosis Autosomal recessive genetic disorder CFTR gene

3. Gene Therapy What is gene therapy? Objective

4. Degree of correction required Minimal percentage of cells with a non-CF phenotyperequired to normalize the functions of the CF bronchial epithelial layer.

5. Barriers for gene delivery Defense mechanisms Delivery systems

8. Adeno-associated

9. Lentivirus

12. AAV vectors

13. Non-viral vectorsResults

14. Conclusions New design of clinical trials New vectors Gene therapy for CF is still a great hope more than a reality

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