20.500.12556/RUL-172870 Pristopi in napredek na področju genskega zdravljenja Approaches and advances in gene therapy Gensko zdravljenje je eden ključnih dosežkov sodobne medicine, saj nam omogoča zdravljenje nekaterih genetskih bolezni. V osnovi gensko zdravljenje pomeni spreminjanje genskega materiala v celicah z namenom zdravljenja ali preprečitve bolezni. To lahko dosežemo z nadomestitvijo okvarjenih alelov ali spremembo izražanja genov. V ta namen se uporablja različne vektorje za vnos nukleinskih kislin v celice, ki so lahko virusni, npr. adenovirusi, AAV, retrovirusi in lentivirusi, ali pa nevirusni, kot so liposomi ali drugi nanodelci. Najnovejše metode temeljijo na uporabi urejanja genoma, predvsem s tehnologijo CRISPR, in ne potrebujejo nujno vnosa eksogenov. Ključni pristopi genskih terapij vključujejo predvsem dodajanje funkcionalnih genov, urejanje genoma in epigenetske modifikacije. V nalogi so predstavljeni ključni zgodovinski dogodki genske terapije, uporaba genskih terapij, etične dileme in ekonomski izzivi. Kljub visokim stroškom razvoja in možnim stranskim učinkom so bile nekatere terapije že odobrene in kažejo zelo obetavne rezultate. Gensko zdravljenje odpira novo poglavje v medicini in predstavlja upanje za številne bolnike. 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. gensko zdravljenje genske terapije gensko inženirstvo vektorji vnosa klinična uporaba etični vidik gene therapy genetic therapies genetic engineering delivery vectors clinical applications ethical considerations true false false Slovenski jezik Angleški jezik Diplomsko delo/naloga 2025-09-12 07:15:14 2025-09-12 07:15:25 2025-10-26 03:49:28 0000-00-00 00:00:00 2025 0 0 0000-00-00 NiDoloceno NiDoloceno NiDoloceno 0000-00-00 0000-00-00 0000-00-00 263631 248747267 51607.pdf 51607.pdf 1 2D7F8BA4585AE6BBC8C9E875065312E9 a776c9535a33fd0b31ff1ff8329090d275db19acbdc6e1446ad3ce74c06ee7aa 622e1fc5-8f97-11f0-9328-0050569b8976 https://repozitorij.uni-lj.si/Dokument.php?lang=slv&id=217004 Biotehniška fakulteta 0 0 0