Subjects

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Gene therapy - GF325

Title:	Genová terapie
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:	unlimited / 34 (unknown)
Min. number of students:	unlimited
4EU+:	no
Virtual mobility / capacity:	no
Key competences:
State of the course:	taught
Language:	Czech
Teaching methods:	full-time
Teaching methods:	full-time
Level:
Note:	course can be enrolled in outside the study plan enabled for web enrollment

Guarantor:	doc. PharmDr. Miroslav Miletín, Ph.D.
Comes under:	4.ročník 2023/24 Farmacie 4.ročník 2023/24 Farmacie (FAR)

Examination dates WS schedule

Annotation -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

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.

Course completion requirements -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

Credit and exam.

Literature -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

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.

Syllabus -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

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.

Teaching methods -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

Lectures and seminars.

Requirements to the exam -

Last update: doc. PharmDr. Miroslav Miletín, Ph.D. (19.09.2023)

Elaborate and in a seminar present a presentation concernig selected topic of gene therapy, answer questions of teacher and other seminar participants.