Primjena genske terapije i matičnih stanica u liječenju oštećenja hrskavice

master's thesis

• Title (english): Application of gene therapy and stem cells in cartilage damage therapy
• Author: Ivan Matolić Galic
• Mentor: Frane Paić (mentor)
• Committee member: Mislav Jelić (predsjednik povjerenstva)
• Committee member: Ljiljana Šerman (član povjerenstva)
• Committee member: Frane Paić (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Medical Biology); Zagreb
• Defense date and country: 2021-07-16, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences

Abstract

U posljednjih nekoliko stoljeća, životni tempo se neprestano ubrzava, a životni vijek je sve duži i stresniji. Prehrana je nezdrava, sve više i više ljudi je pretilo, a razvojem tehnologije razvijaju se i računala i obavljaju sve više posla, što dovodi do toga da znatan broj ljudi radi u uredu ili ima neko slično, sedentarno zanimanje. Sve gore navedeno su faktori rizika za razvoj jedne od
degenerativnih bolesti hrskavice. Liječenje ovih bolesti je kompleksno, a terapijske metode su za sad ili nedovoljno efektivne dugoročno gledano ili pre invazivne u ranoj ili umjereno razvijenoj fazi bolesti. Razvojem regenerativne medicine, došlo do novog pogleda na metode liječenja oštećenja hrskavice jer postoji razlika između tkiva dobivenog popravkom hrskavice i onog dobivenog regeneracijom hrskavice. Cilj je svakako postići regeneraciju hijaline hrskavice, nasuprot cijeljenja fibroznom. Postoji velik broj modela koji se proučavaju kao potencijalne metode liječenja osteoartritisa, degenerativne bolesti diska i patologije meniskusa koje koriste matične stanice, najčešće mezenhimalne matične stanice. Ispituje se primjena matičnih stanica na nosačima, ubrizgavanje istih u zglobnu pukotinu ili pak injiciranje samo njihovih produkata u zglob u obliku egzosoma. Matične stanice mogu biti ubrizgane onakve kakve su izolirane, nepromijenjene ili se može njima manipulirati citokinima i faktorima rasta. Uz matične stanice, potencijal za korištenje u terapiji već navedenih bolesti ima i genska terapije koja se, isto kao i terapija matičnim stanicama, istražuje u raznim oblicima. Dio istraživanja kao vektore koristi viruse, najčešće adenoviruse i rekombinantne adeno-povezane viruse, drugi koriste neviralne vektore. U ovom pregledu literature su navedene činjenice iz trenutno dostupnih radova s tematikom matičnih stanica i genske terapije. Znanstvena zajednica se slaže da je ovo područje istraživanja zanimljivo i obećavajuće, ali se isto tako slaže i da nedostaje velik broj kvalitetnih istraživanja i dobro osmišljenih kliničkih pokusa.

Abstract (english)

During several last centuries, the pace of life is constantly getting faster and life span is getting longer and more stressful. Diet is unhealthy and statistics regarding obesity are catastrophic. Perpetual development of technology has lead to a situation in which most of the work is being done in front of a computer, sitting. Everything that was just stated is a risk factor for the development of degenerative cartilage disease. Treatment of these diseases is complex while therapy methods are either not effective enough in the long run or too invasive for a patient with mild disease. With the development of regenerative medicine, new perspectives concerning therapy methods have emerged because there is a big difference between tissue reparation and restoration. The goal of the treatment method should be hyaline cartilage restoration, as opposed to the reparation with fibrocartilage. There are numerous models of potential osteoarthritis, degenerative disc disease, and meniscus treatments that could be using mesenchymal stem cells in the future. Likewise, there are several methods for stem cell application being tested, such as stem cell scaffolds, injection of the cells in the joint and injection of the products of the stem cells via their exosomes. Stem cells can be used in therapy without any treatment with growth factors or cytokines, but many authors have experimented with the preparation of mesenchymal stem cells with different molecules. Besides stem cells, gene therapy has shown great potential for the treatment of degenerative cartilage diseases. There are quite a few ways of using gene therapy for these diseases. For instance, a portion of researchers uses adenoviruses, while other use recombinant adeno-associated viruses. Others use non-viral methods of gene transfer. The facts listed in this review are collected from scientific researches concerning gene therapy and stem cells. The scientific community agrees on the prosperity and theoretical possibilities of this area of research, but there is also a consensus that states that there is a further need for investigation and well-designed experiments and clinical trials.

• Keywords: matične stanice; genska terapija; hrskavica; osteoartritis; degenerativne bolesti hrskavice; regenerativna medicina
• Keywords (english): stem cells; gene therapy; cartilage; osteoarthritis; degenerative cartilage diseases; regenerative medicine
• Language: croatian
• URN:NBN: urn:nbn:hr:105:876755
• Study programme: Title: Medicine; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: doktor/doktorica medicine (doktor/doktorica medicine)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2022-03-11 10:52:10