Gene Therapy


Offizielle Daten in der Fachveröffentlichung für das folgende akademische Jahr: 2020-2021



Vorlesungen: 12

Praktika: 0

Seminare: 0

Insgesamt: 12


  • Kode des Kurses: OSF-GTH-T
  • 1 kredit
  • Dentistry
  • Optional modul
  • spring


Zahl der Kursteilnehmer für den Kurs:

min. 3 – max. 30

Erreichbar als Campus-Kurs für 30 fő számára. Campus-karok: ÁOK ÁJK ETK KPVK GYTK KTK MK MIK TTK


We wish to give a short overview about the present state of gene therapy, the current ongoing clinical trials and the potential side effects of gene therapy and about the ethical considerations. The various viral and non-viral gene delivery protocols suitable for high efficiency gene delivery both under in vitro and in vivo conditions will be discussed in details. Gene therapy protocols applied in the treatment of malignant diseases will be presented together with the results of ongoing clinical trials. We will talk about the results and the potentials of the treatments of inherited genetic disorders. One of the most promising field of the current clinical trials is the treatment of ischemic diseases, the results will be presented. The potential applications of stem cells during gene therapy will be described, as well. Finally, we will discuss the side effects occurring during gene therapy and the ethical considerations will also be mentioned.


  • 1. Introduction to gene therapy, ongoing clinical trials - Dr. Sáfrány Géza
  • 2. Gene delivery protocols, viral vectors applied in gene therapy - Dr. Sáfrány Géza
  • 3. Targeted gene delivery and targeted gene expression - Dr. Sáfrány Géza
  • 4. Application of dendritic cells in gene therapy - Dr. Sáfrány Géza
  • 5. Gene directed enzyme pro-drug therapy of malignant diseases - Dr. Sáfrány Géza
  • 6. Immune therapy of malignant diseases - Dr. Sáfrány Géza
  • 7. Gene therapy of leukaemia - Dr. Sáfrány Géza
  • 8. Treatment of immune-deficiencies with gene therapy - Dr. Sáfrány Géza
  • 9. Gene therapy of ischemic diseases - Dr. Sáfrány Géza
  • 10. Gene therapy of cystic fibrosis - Dr. Sáfrány Géza
  • 11. Gene therapy of thalassemia - Dr. Sáfrány Géza
  • 12. Oncolytic viruses in tumor therapy - Dr. Sáfrány Géza



Materialien zum Aneignen des Lehrstoffes

Obligatorische Literatur

1. Kelly K. Hunt, Stephan A. Vorburger and Stephen G. Swisher (ed.): Gene Therapy for Cancer, 2007 Humana Press Inc. 999 Riverview Drive, Suite 208 Totowa, New Jersey 07512
2. Thomas F. Kresina (ed.): Introduction to Molecular Medicine and Gene Therapy, 2001 by Wiley-Liss, Inc. ISBNs: 0-471-39188-3 (Hardback); 0-471-22387-5 (Electronic)

Vom Institut veröffentlichter Lehrstoff

1. Sáfrány G.: A génterápia génsebészeti alapjai és jelenlegi helyzete, A Genom (ed.: Hídvégi EJ), Budapest, Széphalom Könyvműhely 2003, pp 187-204
2. Sáfrány G.: A vasculáris génterápia és a csontvelőőssejt-terápia, Atherosclerosis (ed.: Császár A.), Budapest, Synergo Kiadó, 2004 pp 336-344


Empfohlene Literatur

Voraussetzung zum Absolvieren des Semesters

Maximum of 25 % absence allowed



Möglichkeiten zur Nachholung der Fehlzeiten

Joining later lectures, individual consultations.


Multiple choice tests for checking the acquisition of course material is given at the end of semester. Questions include material discussed in lectures and seminars.


  • Dr. Sáfrány Géza

Praktika, Seminarleiter/innen

  • Dr. Sáfrány Géza