Gene Therapy

Data

Official data in SubjectManager for the following academic year: 2024-2025

Topic

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, including mRNA vaccines 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 as well as covid SARS-2 vaccinations. Finally, we will discuss the side effects occurring during gene therapy and the ethical considerations will also be mentioned.

Lectures

• 1. Introduction to gene therapy, ongoing clinical trials - Sáfrány Géza
• 2. Gene delivery protocols, viral vectors applied in gene therapy - Sáfrány Géza
• 3. Targeted gene delivery and targeted gene expression - Sáfrány Géza
• 4. Covid-SARS2 vaccination - Sáfrány Géza
• 5. Gene directed enzyme pro-drug therapy of malignant diseases - Sáfrány Géza
• 6. Immune therapy of malignant diseases - Sáfrány Géza
• 7. Gene therapy of leukaemia - Sáfrány Géza
• 8. Treatment of immune-deficiencies with gene therapy - Sáfrány Géza
• 9. Gene therapy of ischemic diseases - Sáfrány Géza
• 10. Gene therapy of cystic fibrosis - Sáfrány Géza
• 11. Gene therapy of thalassemia - Sáfrány Géza
• 12. Oncolytic viruses in tumor therapy - Sáfrány Géza

Practices

Seminars

Reading material

Obligatory literature

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)

Literature developed by the Department

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

Notes

Recommended literature

http://www.genetherapynet.com/clinicaltrialsgov.html

http://www.bioportfolio.com/cgi-bin/acatalog/Human_Gene_Therapy_Course.html

http://openlearn.open.ac.uk/course/view.php?id=2430

Conditions for acceptance of the semester

-

Mid-term exams

1 Test

Making up for missed classes

Joining later lectures, individual consultations.

Exam topics/questions

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.

Examiners

• Sáfrány Géza

Instructor / tutor of practices and seminars

• Sáfrány Géza