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undergraduate thesis
Primjena genske terapije kod hemoglobinopatija
Zagreb: University of Zagreb, Faculty of Science, 2017. urn:nbn:hr:217:950905
Vujić, Ana
University of Zagreb
Faculty of Science
Department of Biology

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Vujić, A. (2017). Primjena genske terapije kod hemoglobinopatija (Undergraduate thesis). Zagreb: University of Zagreb, Faculty of Science. Retrieved from https://urn.nsk.hr/urn:nbn:hr:217:950905

Vujić, Ana. "Primjena genske terapije kod hemoglobinopatija." Undergraduate thesis, University of Zagreb, Faculty of Science, 2017. https://urn.nsk.hr/urn:nbn:hr:217:950905

Vujić, Ana. "Primjena genske terapije kod hemoglobinopatija." Undergraduate thesis, University of Zagreb, Faculty of Science, 2017. https://urn.nsk.hr/urn:nbn:hr:217:950905

Vujić, A. (2017). 'Primjena genske terapije kod hemoglobinopatija', Undergraduate thesis, University of Zagreb, Faculty of Science, accessed 18 April 2024, https://urn.nsk.hr/urn:nbn:hr:217:950905

Vujić A. Primjena genske terapije kod hemoglobinopatija [Undergraduate thesis]. Zagreb: University of Zagreb, Faculty of Science; 2017 [cited 2024 April 18] Available at: https://urn.nsk.hr/urn:nbn:hr:217:950905

A. Vujić, "Primjena genske terapije kod hemoglobinopatija", Undergraduate thesis, University of Zagreb, Faculty of Science, Zagreb, 2017. Available at: https://urn.nsk.hr/urn:nbn:hr:217:950905

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Metadata
TitlePrimjena genske terapije kod hemoglobinopatija
Title (english)Application of gene therapy in hemoglobinopathies
AuthorAna Vujić
MentorPetra Korać (mentor)
GranterUniversity of Zagreb
Faculty of Science
(Department of Biology)
Zagreb
Defense date and country2017-09-18, Croatia
Scientific / art field,
discipline and subdiscipline		NATURAL SCIENCES
Biology
Abstract
Hemoglobinopatije su jedne od najčešćih monogenskih nasljednih bolesti na svijetu. Dijele se na talasemije i bolesti strukturnih varijanti hemoglobina. U genskoj terapiji kod hemoglobinopatija razlikuju se dva osnovna pristupa: ugradnja globinskih gena i uređivanje globinskih gena i globinskih regulacijskih elemenata. Klinička istraživanja na ljudima su zasad ograničena na pristup ugradnje globinskih gena koji je kod pacijenata s β-talasemijom i srpastom anemijom pokazao pozitivne rezultate. Primjena uređivanja globinskih gena i globinskih regulacijskih elemenata je ograničena na istraživanja na miševima i ljudskim stanicama u kulturi, ali rezultati pokazuju njihovu moguću primjenu u liječenju. U ovom radu dan je kratak pregled osnovnih tehnika koje se koriste u spomenutim pristupima kao i rezultata studija i kliničkih istraživanja koja su dovela do njihovog napretka. Također, izloženi su nedostaci i prednosti oba pristupa te prepreke koje se moraju prijeći da bi genska terapija kao način liječenja hemoglobinopatija postala ostvariva.
Abstract (english)
Hemoglobinopathies are one of the most common inherited monogenic diseases in the world. Two main groups of hemoglobinopathies are thalassemia syndromes and structural hemoglobin variants. In gene therapy for the treatment of hemoglobinopathies there are two main approaches: globin gene addition and gene editing of globin gene and globin regulatory elements. For now, clinical trials on humans are limited on globin gene addition which showed positive results in patients with β-thalassemia and sickle cell anemia. Use of gene editing of globin gene and globin regulatory elements are limited on trials on murine models and human cells in culture. However, results show their possible application in therapeutic purposes. In this paper brief review of basic techniques that are used in both approaches is given as well as description of studies and clinical trials that led to their progress. The main advantages and disadvantages of both approaches and obstacles that must be overcome to make gene therapy the way of treatment for hemoglobinopathies achievable option are also discussed.
Keywords
Keywords (english)
Languagecroatian
URN:NBNurn:nbn:hr:217:950905
Study programmeTitle: Ecology
Study programme type: university
Study level: undergraduate
Academic / professional title: sveučilišni/a prvostupnik/prvostupnica (baccalaureus/baccalaurea) biologije (sveučilišni/a prvostupnik/prvostupnica (baccalaureus/baccalaurea) biologije)
Type of resourceText
File originBorn digital
Access conditionsOpen access
Terms of useLicence In copyright icon
RepositoryRepository of the Faculty of Science
Created on2017-10-19 11:51:42

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