Zusammenfassung
Im Jahre 1868 isolierte Friedrich Miescher bei der Untersuchung von Leukozytenzellkernen eine phosphorhaltige, saure Substanz die er als Nuklein bezeichnete. Obwohl Miescher bereits vermutete, dass diese Nukleinsäure irgendwie im Zusammenhang mit der Vererbung steht, verging mehr als ein dreiviertel Jahrhundert, bis Oswald T. Avery, Colin MacLeod und Maclyn McCarty 1944 den Beweis erbrachten, dass DNA (Desoxyribonukleinsäure) der Träger der genetischen Information ist. Sie fanden heraus, dass DNA, welche aus Bakterien eines virulenten Pneumococcus-Stammes vom Phänotyp „S“ (smooth) isoliert worden war, den nicht virulenten Stamm „R“ (rough) genetisch in dessen virulente und für Mäuse pathogene Form umwandelte (transformierte). Avery und seine Mitarbeiter folgerten daraus, dass DNA (und nicht Protein) die Information für die Virulenz enthielt (Avery et al., 1944). Genaugenommen legten sie mit diesem Experiment nicht nur den Grundstein für die Molekularbiologie und die moderne Biotechnologie, sondern auch für deren jüngsten Spross, die somatische Gentherapie. Immerhin gelang ihnen die Übertragung genetischer Information auf ursprünglich apathogene Bakterien (Streptococcus pneumoniae, Stamm „R“) mit einer damit verbundenen phänotypischen Änderung dieses Stamms in dessen pathogène „S“ Form, welche in der Maus zur Pneumonie führte. Eine ähnliche, wenn gleich therapeutische und auf eukaryonte Zellen ausgerichtete Intention verfolgt auch die Gentherapie. Ziel der somatischen Gentherapie ist nämlich die Übertragung genetischen Materials in die Körperzellen eines Individuums, mit dem Ziel eine therapeutische Wirkung zu erreichen.
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Weiss, L. (2001). Lebergentherapie: Aktueller Stand und Ausblick. In: Raem, A.M., Braun, R.W., Fenger, H., Michaelis, W., Nikol, S., Winter, S.F. (eds) Gen-Medizin. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-56818-3_22
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