Subjects

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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 2022
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.

Examination dates 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. (22.08.2024)

Course completion requirements -

Credit and exam.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (22.08.2024)

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. (22.08.2024)

Teaching methods -

Lectures and seminars.

Last update: Miletín Miroslav, doc. PharmDr., Ph.D. (22.08.2024)

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. (22.08.2024)

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. (22.08.2024)