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PRINCIPLES AND PERSPECTIYES OF GENE THERAPY

Lecturer: Józef Dulak, PhD, DSc Hours and points: 30 h lecture, ECTS 3 Completing reąuirements: colloąium Entry conditions: Course in Biochemistry Scheduled time: Winter semester

The aim of gene therapy is to cure the diseases by influencing on the mechanisms of their origin. Gene therapy is based on delivery of nucie ic acid (DNA or RN A) to cells and organs in order to correct the genetic defect responsible for the disease. Great hopes were created by the animal experiments and the first human trials, but the therapeutic efficiency was often exaggerated. During the course the biological principles of gene transfer will be discussed and their application in several types of diseases will be presented.

Content of the course:

History of gene therapy. Gene transfer in vitro and in vivo. Therapetic genes and marker genes. Vectors. Plasmid vectors: construction and application. Viral vectors: retroviral, adenoviral, adeno-associated viral vectors, helper-dependent vectors. Inhibition of gene expression by nucleic acids: antisense oligonucleotides, DNA decoys, ribozymes. Gene therapy of immmunodeficiency diseases. Gene therapy of other monogenic diseases (cystic fibrosis, Duchenne muscular dystrophy, hemophilia). Gene therapy of cardiovascular diseases: atherosclerosis, hypertension, myocardial infarction, stroke. Pro-angiogenic gene therapy. Gene therapy of cancer: immune gene therapy, suicide gene therapy, anti-angiogenic gene therapy. Cell-based gene therapy: therapeutic potentials of stem cells. Gene therapy and cloning. Ethical aspects of gene therapy.