Educational guide 2018_19 Faculty of Oenology

Contents

Topic	Sub-topic
1. Introduction. Molecular Fundamentals of Gene Therapy:	Molecular bases of genetic diseases, scientific basis for the development of gene therapy, administrative regulation for the approval of the use of biological drugs (FDA).
2. Modulation of the gene function using nucleic acid molecules	DNA - Aptames, chimeroplasts (gene repair), SHFR, Oligonucleotides (SSO, LNA, TFO), homologous recombination (gene repair); RNA - Spliceosoma-mediated RNA trans-splicing (SMaRT), antisense oligonucleotides, Ribozims.
3. Gene Therapy Vectors:	Non-viral - naked DNA, liposomes, lipoplexes, polyplexes; Viral - Adenovirus, Retrovirus, Adeno-associated virus, Lentivirus, Herpes virus.
4. Limitations of gene transfer:	Immune response, anatomical and cellular barriers, etc.
5. Regulation of the expression of the transgene	Episomal replication, 3 '5' regulatory sequences, ceiling induction systems on/off.
6. Applications of Gene Therapy techniques	Gene therapy of hereditary diseases (monogenic versus multigens), Gene therapy of degenerative diseases and CNS, Gene therapy of acquired diseases (cancer, infectious).
7. Ethics and gene therapy:	Public perception of the risks associated with the use of molecular medicine both the individual (concomitant alteration of gene expression) and the population.
8. Future and gene therapy	Future prospects and desirable consequences.