Syllabus
Lectures:
? Definition of gene therapy (GT).
? Basic requirements and potential.
? Current state.
? Division: o based on application way:
? in vivo
? ex vivo o based on targeted cells type:
? GT of somatic cells
? GT of stem cells o based on technology used:
? vector mediated GT
? oligonucleotide mediated GT
? gene immunisation
? vector mediated GT o ways of vector mediated o GT of cancer o characterisation of vectors:
? DNA in complex
? naked DNA
? viral vectors
? oligonucleotide mediated GT o antisense approach
? principles of mechanism of action
? modifications of antisense oligonucleotides
? examples of practical use o antigene approach (triplex forming oligonucleotides, TFO)
? mechanism of action
? modifications of antigene oligonucleotides
? examples of practical use
? Chemical structure, modifications and methods of synthesis of antisense, antigene a fluorescently labelled oligonucleotides
? RNA interference o synthetic siRNA o siRNA produced of s vector o knock-down vs. knock-out
? Gene (genetic) immunisation o principle o examples of practical use
? Tailor made medicines o prerequisites of genetic testing o ethical risks
? Biotechnology base drugs o Research on the new BIO-drugs field o biotechnological generics ? so-called ?biosimilar drugs?
Tutorials:
? Antisense and antigene therapeutics.
? Practical show of automated oligonucleotide synthesis.
? Prezentations of students on selected topics.
Annotation
In the course of the subject lectures students learn the basic principles and scope of gene therapy, the methods employed in gene therapy and interferences between gene therapy and pharmacy. Tutorials are be focused on discussion about main topics of gene therapy, its current and potential fields of application, and a demonstration of oligonucleotide synthesis on a automated RNA/DNA synthesizer.
Students present their presentations in a selected area.