Niewirusowe preparaty genowe – charakterystyka metod oceny właściwości fizykochemicznych

• Authors: Emilia Kuźnicka , Maciej Małecki

Title variants

EN

Non-viral gene preparations – methods of evaluation of physico-chemical properties

• Languages of publication: PL

Abstracts

PL

Stanowiące podstawę terapii genowej geny kodujące terapeutyczne białka wprowadzane są do komórek za pomocą nośników wirusowych i niewirusowych. Brak jednoznacznie bezpiecznych, a jednocześnie efektywnych metod wprowadzania genów sprawia, iż zastosowanie terapii genowej w klinice jest wciąż ograniczone. Prowadzone są intensywne badania poświęcone wektorom niewirusowym – nośnikom genów, które wydają się stabilne in vitro i bezpieczne dla pacjentów. Kompleksy molekularne plazmidowego DNA (pDNA) i związków polimerowych tworzą się spontanicznie, w wyniku oddziaływań elektrostatycznych. Sposób oddziaływania pDNA ze związkami polimerowymi wpływa na właściwości fi zykochemiczne kompleksów pDNA : wektor kationowy, które mają ogromne znaczenie dla efektywności wprowadzania genów do komórek (transfekcji). Wdrażanie nowych, aktywnych biologicznie preparatów genowych do badań eksperymentalnych wiąże się z koniecznością prowadzenia prac poświęconych ocenie ich właściwości fi zykochemicznych i biochemicznych, które wykonuje się metodami stosowanymi w laboratoriach inżynierii genetycznej; często szuka się również nowych rozwiązań analitycznych. W artykule podjęto próbę scharakteryzowania niewirusowych preparatów genowych głównie w kontekście oceny ich właściwości fizykochemicznych. Opisano aktualnie stosowane metody badania preparatów genowych. Świadomość możliwości prowadzenia w pracowniach różnych dyscyplin naukowych badań właściwości kompleksów pDNA : : nośnik może pomóc w projektowaniu nowych preparatów genowych i próbach ich wykorzystania w leczeniu chorych.

EN

Gene therapy is based on the use of viral and non-viral nucleic acid preparations. They allow to administration of the therapeutic genes into targeted cells. Due to the lack of safe and eff ective methods of gene delivery, use of gene therapy in the clinic is restricted. The large attention is focused on studies of non-viral vectors, which seem to be stable and safe gene vehicles. Molecular complexes between plasmid DNA and cationic polymers, such as cationic lipids, polypeptides are formed spontaneously, as a result of electrostatic interactions. The mode of interaction of pDNA with polymeric compounds has a strict infl uence on physico-chemical properties of pDNA : cationic vector complexes. It is closely related to the gene transfer effi ciency (transfection). The evaluation of physical and molecular mechanisms of complex formation is crucial for effi cient gene administration into the cells. In this article various methods of studies of physicochemical properties of pDNA : vehicle complexes are described with special attention to their experimental/clinical value.

Keywords

EN

cationic polymers; kompleksy pDNA : nośnik; niewirusowe preparaty genowe; non-viral gene preparations; pDNA : vehicle complexes; polimery kationowe; transfection; transfekcja

Discipline

• MEDICINE: MEDICINE

• Publisher: Śląski Uniwersytet Medyczny w Katowicach
• Journal: Annales Academiae Medicae Silesiensis
• Year: 2011
• Volume: 65
• Issue: 5-6
• Pages: 67–79

Contributors

author

Emilia Kuźnicka

• Zakład Biologii Komórki Centrum Onkologii – Instytut im. Marii Skłodowskiej-Curie w Warszawie

author

Maciej Małecki

• Zakład Biologii Komórki Centrum Onkologii – Instytut im. M. Skłodowskiej-Curie w Warszawie ul. Roentgena 5 02-781 Warszawa tel. [phone]

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• Document Type: article