Gene Therapy - GAPS325
Title: Gene Therapy
Guaranteed by: Department of Pharmaceutical Chemistry and Pharmaceutical Analysis (16-16190)
Faculty: Faculty of Pharmacy in Hradec Králové
Actual: from 2025
Semester: winter
Points: 0
E-Credits: 2
Examination process: winter s.:oral
Hours per week, examination: winter s.:14/14, C+Ex [HS]
Capacity: unknown / unknown (unknown)
Min. number of students: unlimited
4EU+: no
Virtual mobility / capacity: no
Key competences:  
State of the course: taught
Language: English
Teaching methods: full-time
Level:  
Is provided by: GAF325
Note: course can be enrolled in outside the study plan
enabled for web enrollment
Guarantor: doc. PharmDr. Miroslav Miletín, Ph.D.
Comes under: 2.ročník 2024/25 PS povinně volitelné
WS schedule   
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.
Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)
Course completion requirements -

Credit and exam.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)
Literature -

Recommended:

  • Daniel Scherman . Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology. Paris: National Scientific Research Center (CNRS), France, 2014, s. ISBN 978-1-84816-828-2.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)
Teaching methods -

Lectures and seminars.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)
Requirements to the exam -

To elaborate and present a short overwiev about a selected topic at a seminar, answer questions of the teacher and the seminar participants.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)
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.

 

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (24.09.2025)