Download Gene Therapy

GENE THERAPY
The aim of the subject is to present the basic information about genetic engineering applied to the
practical problems, including attempts to treat diseases with the means of gene therapy (GT) and
immune gene therapy. The theoretical principles. presented at the time of lectures involve
definitions, strategies, techniques and tools of GT, examples of treated diseases as well as clinical
trials. The practical tutorials are arranged in order to acquaint students with basic laboratory
methods of GT, with special attention to plasmid amplification, isolation, target cell transfection and
assessment of lab procedures efficacy. The skills previously acquired during the classes of
biochemistry, genetics and immunology are useful for student better understanding GT issues.
TEACHERS:
 dr hab. Piotr Kopiński
 mgr Joanna Wielikdzień
CONTACT: [email protected]
SYLABUS
I.
Name of the unit offering the course: Department of Gene Therapy
II.
Head of the Unit:/ course coordinator dr hab. Piotr Kopiński
III.
2nd year, number of hours: 10
IV.
Form of classes” lectures: 4, tutorials: 6
V.
Form of the crediting: credit with grade
VI.
Number of ECTS points: 2
VII.
Topics:
Lectures (ahead of tutorials):
Basic concepts and issues of GT. Techniques of treatment in vivo and ex vivo. Viral vectors and
plasmids as the tools of GT. Human cells in genetic engineering, examples. Antisense
oligonucleotides, siRNA and aptamers in GT. GT target genes, examples. The concept of
immune gene therapy. Examples of clinical trials using GT techniques in oncologic and
circulatory diseases.
Tutorials (6 hours, Department laboratory):
Principles of culture of human established cell lines. Plasmid construction, examples of shRNA
plasmids. Transformation of competent bacteria and amplification of genetic material. Plasmid
isolation from bacterial cultures. Transfection of selected tumor cell lines – use of shRNA
encoding vector as an example. Efficacy examination of target oncogene expression silencing
with use of RT-PCR technique.
VIII. Booklist:

Giacca M: Gene Therapy. Springer-Verlag, Mediolan 2010 selected topics, coppies
avilable in the Department
RULES AND REGULATIONS
1. General rules:
a. tutorials are obligatory, students presence at tutorials is registered,
b. the scope of knowledge required for each tutorial is given on Department's information
board,
c. students comprehension of material can be checked at the tutorial,
2. Form and conditions of course completion:
a. written test covering topics of both lectures and tutorials,
b. the test consists of 30 questions. In terms of passing the exam student has to obtain the
score of more than 50%.
c. the final test can take place only after the last tutorial,
d. in case of failed test, second attempt can take place, its date must be accepted by the
lecturer,
e. failure of the second attempt results in board exam,
f. unjustified absence during any test is regarded as a failed test.
3. Criteria of the course completion:
a. participation in all tutorials,
b. mastering the knowledge required for tutorials and lectures,
c. mastering the knowledge included in indicated literature.
4. Criteria of justifying the absence:
a. every absence during tutorials must be excused at the first tutorials after the absence,
b. in case of justified absence student has to learn the material discussed during the missed
tutorials and the student's comprehension of the material has to be checked within two
weeks. It is student's responsibility to make an appointment with the teacher.
c. there is a possibility to comprehend the material discussed during missed tutorials, but it
must occur before the final test.