Aktuelle Entwicklung der Gentherapie bei pädiatrischen Erkrankungen. Molekulare Präzisionsmedizin als kausale Therapie bei angeborenen und erworbenen Erkrankungen

Rosenecker, J. (2020): Aktuelle Entwicklung der Gentherapie bei pädiatrischen Erkrankungen. Molekulare Präzisionsmedizin als kausale Therapie bei angeborenen und erworbenen Erkrankungen. In: Monatsschrift Kinderheilkunde, Bd. 168, Nr. 1: S. 28-38

Abstract

Gene therapy as a treatment concept for human diseases has become accepted in clinical medicine in recent years. To date six gene therapeutic agents have been approved in Europe. Additional gene therapeutic agents, which have already been approved in the USA, are close to approval in Europe. Through gene therapy, oncological immunotherapy procedures could be improved or made possible for the first time. Causal therapies for numerous congenital disorders are being developed. Gene therapies for adenosine deaminase deficiency and beta thalassemia are already available. The large number of clinical gene therapy studies currently in progress and are already in phase II/III, are expected to lead to the approval of additional gene therapeutic agents in the near future. The severe unexpected side effects experienced in the initial gene therapy trials could be avoided by improved gene vector systems. Nevertheless, patients treated by gene therapy are closely monitored because certain viral gene vectors can lead to insertional mutagenesis. In addition to gene replacement therapy, through the development of so-called gene scissors new therapy concepts are possible, which make future genome editing possible up to the correction of individual mutations.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0026-9298
• Sprache: Deutsch