diplomsko delo
Katja Jensterle (Author), Uroš Petrovič (Mentor), Uroš Petrovič (Thesis defence commission member), Polona Jamnik (Thesis defence commission member), Damjana Drobne (Thesis defence commission member)

Abstract

CRISPR-Cas9 je metoda za urejanja genoma, ki bi se potencialno lahko uporabljala v namene genske terapije ljudi. V delu je splošno opisana metoda in navedenih je tudi nekaj dejanskih primerov. Tehnologija urejanja genoma veliko obeta na področju raziskav in zdravljenja genskih bolezni. Do nedavnega se je CRISPR-Cas9 sistem uporabljal klinično le ex-vivo, zdaj pa je znan tudi primer možnosti in vivo uporabe. Metoda CRISPR-Cas9 je uporabna za številne namene, kot so genska terapija, izdelava gensko modificiranih živalskih modelov za preučevanje patologije bolezni človeka, za hkratne tvorbe mutacij na več mestih genoma, za fleksibilne manipulacije epigenoma ter v namene regenerativne medicine. Pri genski terapiji ločujemo zarodno in somatsko terapijo. Zarodna terapija se za razliko od somatske terapije, zaradi etičnih dilem in premalo znanstvenih podatkov klinično ne izvaja, potekajo pa že raziskave na laboratorijskem nivoju. Gre za dokaj etično sporno temo, ki še nima dovolj urejene regulative. Namen diplomskega dela je tudi predstaviti etična vprašanja, s katerimi se srečujemo pri možni uporabi metode CRISPR-Cas9 pri ljudeh.

Keywords

CRISPR-Cas9;somatska terapija;zarodna terapija;genomsko modificiranje;DNA;

Data

Language: Slovenian
Year of publishing:
Typology: 2.11 - Undergraduate Thesis
Organization: UL BF - Biotechnical Faculty
Publisher: [K. Jensterle]
UDC: 606:616-056.7:602.8(043.2)
COBISS: 9282169 Link will open in a new window
Views: 1336
Downloads: 394
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Other data

Secondary language: English
Secondary title: ǂThe ǂuse of CRISPR-Cas9 in gene therapy in humans
Secondary abstract: CRISPR-Cas9 is a method for genome editing, which could potentially be used for the purpose of gene therapy in humans. Here I am going to generally describe the method and list a few examples of its application. The CRISPR technology is a great prospect in the field of research and treatment of genetic diseases. Up until now, the CRISPR-Cas9 method was only clinically used ex-vivo. But lately, there are more and more possibilities of its in vivo use. CRISPR-Cas9 system is applicable for a wide range of purposes such as gene therapy, the creation of genetically modified animal models for the study of the human disease's pathology, making multiple mutations in genome, increasing the manipulation of the epigenome and for the purposes of regenerative medicine. There are two types of gene therapy with CRISPR-Cas9: somatic and germline therapy. In comparison with somatic therapy, the germline therapy is currently forbidden because of ethical dilemmas and a lack of scientific data. The current only use is on the level of research. Germline therapy is an ethically controversial topic, for which adequate regulation still does exits. The purpose of this thesis is also to present the ethical issues that we are faced with the potential use of the CRISPR-Cas method.
Secondary keywords: somatic therapy;germline therapy;genome modification;
Type (COBISS): Bachelor thesis/paper
Study programme: 0
Embargo end date (OpenAIRE): 1970-01-01
Thesis comment: Univ. v Ljubljani, Biotehniška fak., Študij biotehnologije
Pages: VI, 23 str.
ID: 11212354