Syllabus
Overview of gene therapy and legislation
Divisions of gene therapy.
Legislation in area of gene therapy and genetically modified organisms.
Methods of preparation of bio-oligomers of nucleic acids type
Principles of chemical synthesis of oligopeptides and nucleic acids (oligonucleotides) on solid phase.
Automatized chemical synthesis of oligonucleotides, synthesizers.
Modifications of oligonucleotides for therapeutic and diagnostic use.
Oligonucleotide therapeutics
Types of oligonucleotide therapeutics
Antisense therapeutics
SiRNA
DNAzymes
Oligonucleotide aptamers
Preparations in advanced phases of clinical trials.
Other therapeutic and diagnostic potential of synthetic nucleic acids (drug delivery, aptasensors, etc.)
RNA interference as gene therapy tools
Overview of non-coding RNA.
MicroRNA and SiRNA.
Possibilities of therapeutic use of non-coding RNA.
MicroRNA and SiRNA in clinical trials.
Vector mediated gene therapy (VMGT)
Possibilities of VMGT.
Vector types used in gene therapy.
Ways of vectors preparation.
Advantages and disadvantages of various vectors.
Problems and risks of VMGT.
DNA and RNA vaccines.
DNA and RNA vaccines
Possible excursion at biotechnological company.
Annotation
Gene therapeutics belong among biological drugs. After years of intensive research and expectations, currently therapeutic tools or methods influencing genetic information enter clinical praxis.
Lectures of subject Gene Therapy bring supplementary information to the essential ones presented in course of subject Biological Drugs, focus on the most promising preparations and methods currently entering the clinical praxis or advanced phases of clinical trials. The most important trends in gene therapeutics development are discussed.