aFAAT1_16 Advanced Therapy Medicinal Products

Farmaceutická fakulta
podzim 2025

Předmět se v období podzim 2025 nevypisuje.

Rozsah
2/0/0. 28p. 3 kr. Ukončení: zk.
Vyučováno kontaktně
Vyučující
PharmDr. Jakub Treml, Ph.D. (přednášející)
Garance
PharmDr. Jakub Treml, Ph.D.
Ústav molekulární farmacie – Ústavy – Farmaceutická fakulta
Omezení zápisu do předmětu
Předmět je určen pouze studentům mateřských oborů.
Mateřské obory/plány
Cíle předmětu
Students in this subject will get an overview knowledge about current possibilities in therapies with nucleic acids and also with advanced therapy medicinal products (ATMP), with an emphasis on gene therapy. Special lectures will be dedicated to the legal status of ATMP in EU and USA. Moreover, apart from possible therapies and novel medicinal products, also lectures will touch the topic of their preparation and manufacturing.
Výstupy z učení
Basic knowledge in novel advanced therapy medicinal products and nucleic acid therapy.
Osnova
  • - Overview of advanced therapy and therapy with nucleic acids – basic definitions and general principles
  • - History of advanced therapy
  • - Oligonucleotides – preparation and therapeutic potential
  • - RNA interference (miRNA, siRNA) as a tool in gene therapy
  • - Vectors and gene delivery techniques
  • - Non-viral nucleic acid delivery
  • - Examples of tissue engineering and cellular therapy
  • - Therapeutic options of gene therapy in cancer (e.g. CAR-T)
  • - Therapeutic options of gene therapy in neurological and muscular diseases
  • - Therapeutic options of gene therapy in haemophilia and other diseases
  • - DNA and RNA vaccines
  • - Methods of genetic engineering (CRISPR-Cas9)
Literatura
    doporučená literatura
  • NÓBREGA, Clévio David Rodrigues; Liliana MENDONÇA a Carlos A. MATOS. A handbook of gene and cell therapy. Cham: Springer, 2020, xi, 188. ISBN 9783030413323. info
  • Advanced textbook on gene transfer, gene therapy and genetic pharmacology : principles, delivery and pharmacological and biomedical applications of nucleotide-based therapies. Edited by Daniel Scherman. Second edition. New Jersey: World Scientific, 2020, xv, 617. ISBN 9781786347053. info
  • GIACCA, Mauro. Gene therapy. Dordrecht: Springer, 2010, xxi, 303. ISBN 9788847016422. info
Výukové metody
Lecture Monologic (reading, lecture, briefing)
Metody hodnocení
Written exam
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