Syllabus
I. General partPrinciples, mechanisms and possibilities of gene therapy; ethical problemsTypes of therapeutic nucleic acids (NA) - genes encoding proteins - non-coding NA (oligonucleotides, catalytic NA, regulatory NA, aptamers)Delivery of genetic material - viral (retroviruses, adenoviruses, herpetic viruses, adeno-associated viruses) - non-viral - physical (electroporation, sonoporation, biolistic delivery) - chemical (liposomes, polymers)Gene editing
II. Gene therapy of tumorsRegulation of gene expressionImmunotherapy - modified T lymphocytes (TCR, CAR) - vaccines - DNA and RNA vaccines, recombinant viruses, cell vaccinesSuicide genesOncolytic viruses
III. Gene therapy of non-malignant diseases – student presentationsHematopoietic stem cellsNeurodegenerative diseasesMuscle dystrophyCardiac diseasesViral infectionsOcular diseasesLiver diseasesLung diseases
Annotation
Gene therapy is a discipline striving for the application of up-to-date knowledge and methods into a clinical practice. It comprises methodological approaches of molecular biology and genetic engineering and also understandings from genetics, immunology, biochemistry, virology, oncology, and other biological and medical fields.
In a general part of the course, principles, mechanisms, and potential usage of gene therapy will be explained as well as gene transfer and ethical problems associated with gene therapy. Another part of the course will be focused on the utilization of gene therapy in oncology and the description of main anti-tumor therapeutic procedures.
Finally, students will prepare short presentations (about 10 min) about the development of gene therapy for selected non-malignant diseases.