Educational guide Faculty of Chemistry |
english |
Double degree in Biotechnology and Biochemistry and Molecular Biology 2014 |
Subjects |
GENE THERAPY |
Contents |
IDENTIFYING DATA | 2018_19 |
Subject | GENE THERAPY | Code | 13904205 | |||||
Study programme |
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Cycle | 1st | |||||
Descriptors | Credits | Type | Year | Period | ||||
3 | Optional | 2Q |
Competences | Learning outcomes | Contents |
Planning | Methodologies | Personalized attention |
Assessment | Sources of information | Recommendations |
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. |