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Gene Therapy for the Treatment of Cystic Fibrosis
Stephanie M. Delgado
RISE ProgramBIOL 3095
Cystic Fibrosis Autosomal recessive genetic
disorder CFTR gene
Gene Therapy
What is gene therapy? Objective
Degree of correction required
Minimal percentage of cells with a non-CF phenotype required to normalize the functions of the CF bronchial epithelial layer.
Barriers for gene delivery Defense mechanisms Delivery systems
Vectors Vectors
Target: Airway EpitheliumObjective: Generation of a functional protein
Vectors Viral vectors
AdenoviralAdeno-associatedLentivirusPoxvirus, Sendai, Herpes virus
Vectors
Non-viral vectorsSynthetic vectorsLess likely to induce immune response
Clinical studies
Routes Trial Design
Adenovirus vectorsAAV vectorsNon-viral vectors
Results
Conclusions
New design of clinical trials New vectors Gene therapy for CF is still a great
hope more than a reality
References
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