Gene Therapy
1655-Lek22GENO-J
The lectures are designed to acquire and consolidate knowledge of basic concepts and issues related to gene therapy and immunotherapy: acquiring basic knowledge of in vivo and ex vivo treatment techniques, viral vectors and plasmids as tools for gene therapy; the use of human cells in genetic engineering; learning about techniques using antisense oligonucleotides, siRNA, aptamers in gene therapy, learning about genes modified by gene therapy - examples and specific diseases. In addition, lectures include the presentation of examples of clinical trials using gene therapy techniques in the treatment of oncological and cardiovascular diseases.
Seminars will familiarize students with selected gene therapy methods in a clinical context and present gene immunotherapy methods applied to practical problems, with particular focus on adoptive immune therapy methods, i.e., CAR-T and the CRISPR-Cas9 method. In addition, the seminars are devoted to acquiring the ability to design simplified experiments using gene therapy protocols to treat selected diseases.
Exercises are devoted to acquiring practical skills in selecting and applying gene therapy techniques. Students carry out culture of established and primary human cell lines; they learn the principles of RNA interference and perform transfection of selected cancer cell lines using sequences that inhibit the expression of target oncogenes and evaluate the efficiency of the technique. They carry out isolation of mononuclear cells to establish cocultures with cancer cells to obtain a hybrid that forms the basis of an anti-cancer vaccine.
Total student workload
1.Workload associated with direct participation of academic teachers:
- lectures: 5 h
- seminars: 12 h
- tutorials: 8 h
- final test: 1,5 h
Total workload involving the direct participation of academic teachers: 26,5 h, which equals 1,06 of an ECTS point.
2.Total student workload:
- lectures: 5 h
- seminars: 12 h
- tutorials: 8 h
- preparation for tutorials (including reading of the selected literature and written tasks completion): 5 h
- preparation for final test and final test: 6 + 1,5 = 7,5 h
Total student workload: 37,5 h, which equals 1,5 ECTS point.
3. Workload related to achievement of learning outcomes in medical simulation settings (group C): not applicable
4. Workload associated with achievement of learning outcomes related to medical communication: not applicable
Learning outcomes - knowledge
W1: Describes the genetic and epigenetic mechanisms of oncogenesis; Explains the relationship to clinics and therapeutic strategies (C.W9, C.W33)
W2: Characterizes advanced gene therapy methods, techniques and indications (C.W33)
W3: Explains the mechanisms of gene therapy tools in clinical examples (C.W33).
W4: Lists drugs constructed through gene therapy for conventional therapy and indications for their use (C.W9, C.W33).
W5: Explains the mechanisms of action of vaccines and the basics of reprogramming T cells to become a tool for cancer gene immunotherapy (C.W9, C.W33).
W6: Outlines the advantages, disadvantages and risks of gene therapy (C.W33).
W7: Describes the most promising prospects for gene therapy and immunotherapy using CAR-T and CRISPR approaches (C.W9, C.W33).
Learning outcomes - skills
U1: Correctly selects therapeutic targets for gene therapy citing published scientific studies (B.U10, C.U9, C.U12).
U2: Plans and chooses an appropriate gene therapy method or gene immunotherapy technique (C.U9, C.U12).
U3: Appropriately classifies and interprets test results and makes inferences about the effectiveness of gene therapy transfection techniques (B.U10)
U4: Uses appropriate databases to search for studies on current gene therapy trials (C.U12)
U5: Designs simple gene therapy experiments (C.U9)
U6: Determines the risks of using a selected gene therapy (C.U9)
Learning outcomes - social competencies
K1: Attempts to resolve moral and ethical dilemmas (K_K04)
K2: Demonstrates the habit of self-education by participating in laboratory experiments and develops good group work habits (K_K5)
Teaching methods
Lectures:
- informative lecture
- problem lecture
Seminars:
- didactic discussion
- group work
- individual work
- computer-assisted teaching, such as medical databases
Tutorials:
- practical/laboratory exercises
- case study
- didactic discussion
- individual work
- group work
Expository teaching methods
- participatory lecture
- description
- informative (conventional) lecture
Exploratory teaching methods
- laboratory
- project work
- practical
- seminar
Online teaching methods
- content-presentation-oriented methods
- methods referring to authentic or fictitious situations
- exchange and discussion methods
- cooperation-based methods
Type of course
compulsory course
Prerequisites
Students beginning their Gene therapy course should have knowledge and skills previously acquired in molecular biology, biochemistry, and the basics of medical genetics classes.
Course coordinators
Assessment criteria
Final written test (≥60%): W1-W7, U2, U6,
Oral test (>75%): W4, W5, W7, U1, U3,
Report (> 75%): W2, W3, U5
Task completion (>75%): U1-U6
Extended observation (> 50%): K1 - K2
Practical placement
Additional information
Additional information (registration calendar, class conductors,
localization and schedules of classes), might be available in the USOSweb system: