Gene therapy is one of the most important achievements of modern medicine, as it enables the treatment of certain genetic diseases. In gene therapy, the genetic material in cells is modified in order to treat or prevent diseases. This can be achieved by replacing defective alleles or altering gene expression. For this purpose, various vectors are used to introduce nucleic acids into the cells. These can be viral vectors, e.g., adenoviruses, AAV, retroviruses, and lentiviruses, or non-viral vectors, such as liposomes or other nanoparticles. The latest methods are based on the use of genome editing, in particular CRISPR technology, and do not necessarily require the introduction of exogenous genetic material. The most important approaches in gene therapy include the addition of functional genes, genome editing, and epigenetic modifications. In this thesis, the most important historical events in gene therapy, the application, the ethical dilemmas, and the economic challenges of gene therapies are presented. Despite high development costs and possible side effects, some therapies have already been authorised and show promising results. Gene therapy is opening a new chapter in medicine and offers hope for many patients.
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