Plazmidy jako wektory do terapii genowej

Plasmids – vectors for gene therapy
P. Zaleski, P. Wawrzyniak, A. Sobolewska, G. Płucienniczak


1. Wstęp. 2. Naturalna modyfikacja DNA jako przeszkoda w stosowaniu plazmidów w terapii genowej. 3. Bezpieczeństwo użycia plazmidowego DNA. 4. Wprowadzenie pDNA do komórek eukariotycznych. 5. Los plazmidowego DNA po wprowadzeniu do komórek eukariotycznych. 6. Terapie genowe bazujące na pDNA. 7. Inne kierunki rozwoju terapii genowych opartych na plazmidowym DNA. 7.1 Baktofekcja. 7.2. Alternatywna terapia genowa (Alternative Gene Therapy – AGT). 7.3. Hydrożele. 7.4. Minikoliste DNA. 7.5. Mininici DNA. 8. Podsumowanie

Abstract: The first confirmed transfer of genetic material in human was performed in 1990. Ever since, gene therapy was considered to be one of the best promising treatments of genetic diseases. The sine qua non of successful gene therapy are efficient genetic vectors. Recently, the most frequently used vectors in clinical trials for genetic therapies are virus-based and plasmid-based. A range of features makes plasmids useful for gene therapy, however, they have also some characteristics which make it difficult to consider plasmids as ideal vectors. The main goal of this article is to address and describe these unfavourable factors.

1. Introduction. 2. Natural modification of DNA as an obstacle to the use of plasmids for gene therapy. 3. Plasmid DNA usage safety. 4. Plasmid DNA entry into eucaryotic cells. 5. Post-entry fate of plasmid DNA in eucaryotic cells. 6. pDNA-based gene therapies. 7. Alternative routes of development of pDNA-based gene therapies. 7.1. Baktofection. 7.2. Alternative Gene Therapy – AGT. 7.3. Hydrogels. 7.4. DNA minicircles. 7.5. DNA ministrings. 8. Summary