IDENTIFYING DATA 2019_20
Subject (*) GENE THERAPY Code 19204210
Study programme
Bachelor's Degree in Biotechnology (2009)
 Cycle 1st
Descriptors Credits Type Year Period
3 Optional
Language
Català
Department Biochemistry and Biotechnology
Coordinator
RODRÍGUEZ CALVO, RICARDO
 E-mail ricardo.rodriguez@urv.cat
Lecturers
RODRÍGUEZ CALVO, RICARDO
Web
General description and relevant information The general objective is to give students an overview of the advances in the design and application of new biomolecules, which, in contrast to traditional drugs, already allow the development of very specific therapies in comparison with molecular targets. In particular, we will provide them with the tools to understand the conceptual and methodological bases of the new gene therapy strategies that allow correcting in situ genetic defects, together with a critical and exhaustive analysis of the most updated experimental tools and strategies. We will also try to promote interest in the substantive discussion on the scientific and ethical bases of the future molecular medicine and we will encourage its critical spirit to confront the latest advances.

Competences
Type A Code Competences Specific
 A4 Know and understand in an integrated manner the organisms at molecular, cellular and metabolic level.
 A9 Know in depth the microorganisms, both prokaryotes and eukaryotes, viruses, as well as the diversity of metabolisms present in prokaryotes, and their possibilities of biotechnological use.
 A11 Proper application of the diversity of recombinant DNA techniques and methodologies to design cellular genetic engineering strategies for the production of homologous or heterologous proteins or cells capable of acting as biocatalysts
 A13 Identify the possibilities of transferring biotechnology based on basic experimentation and the scaling of processes taking into account the economic parameters, rationalizing the use of matter and energy, and minimizing environmental impacts
 A15 Know the diversity of existing biotechnological processes and products, as well as the recent biotechnological advances, and know how to explain them and communicate them to diverse audiences.
Type B Code Competences Transversal
Type C Code Competences Nuclear

Learning outcomes
Type A Code Learning outcomes
 A4 Understand the conceptual and methodological bases of the new strategies of gene therapy that allow correcting genetic defects "in situ".
Discuss the scientific and ethical foundations of future molecular medicine and foster a critical spirit in the face of the latest advances.
 A9 Describe the advances in the design and application of new biomolecules as opposed to traditional drugs.
 A11 Understand the conceptual and methodological bases of the new gene therapy strategies that allow to correct genetic defects "in situ".
Discuss the scientific and ethical bases of future molecular medicine and foster a critical spirit in the face of the latest advances
 A13 Describe the advances in the design and application of new biomolecules as opposed to traditional drugs.
 A15 Analyze and develop in public the state of the art of an application/technique in the field of gene therapy.
Type B Code Learning outcomes
Type C Code Learning outcomes

Contents
Topic Sub-topic
1. Introduction. Molecular Fundamentals of Gene Therapy: Molecular bases of genetic diseases, scientific basis for the development of gene therapy, administrative regulation for the approval of the use of biological drugs (FDA).
2. Modulation of the gene function using nucleic acid molecules DNA - Aptames, chimeroplasts (gene repair), SHFR, Oligonucleotides (SSO, LNA, TFO), homologous recombination (gene repair); RNA - Spliceosoma-mediated RNA trans-splicing (SMaRT), antisense oligonucleotides, Ribozims.
3. Gene Therapy Vectors: Non-viral - naked DNA, liposomes, lipoplexes, polyplexes; Viral - Adenovirus, Retrovirus, Adeno-associated virus, Lentivirus, Herpes virus.
4. Limitations of gene transfer: Immune response, anatomical and cellular barriers, etc.
5. Regulation of the expression of the transgene Episomal replication, 3 '5' regulatory sequences, ceiling induction systems on/off.
6. Applications of Gene Therapy techniques Gene therapy of hereditary diseases (monogenic versus multigens), Gene therapy of degenerative diseases and CNS, Gene therapy of acquired diseases (cancer, infectious).
7. Ethics and gene therapy: Public perception of the risks associated with the use of molecular medicine both the individual (concomitant alteration of gene expression) and the population.
8. Future and gene therapy Future prospects and desirable consequences.

Planning
Methodologies  ::  Tests
  Competences (*) Class hours
 Hours outside the classroom
 (**) Total hours
Introductory activities
1 0 1
Lecture
A4
A13
A15
 24 28 52
Seminars
6 10 16
Personal attention
1 0 1
 
Extended-answer tests
A4
A13
A15
 2 2 4
Oral tests
1 1 2
 
(*) On e-learning, hours of virtual attendance of the teacher.
(**) The information in the planning table is for guidance only and does not take into account the heterogeneity of the students.

Methodologies
Methodologies
  Description
Introductory activities Introduction on the organization of the subject. Number of topics. Development. Evaluation.
Lecture Theoretical development in the classroom of the content of the subject.
Seminars Global discussion (students + teacher) of an article related to the content of the subject.
Personal attention Individual resolution of doubts.

Personalized attention
Description
The personalized attention will be carried out in office 106. Please request a previous appointment by email: miquel.mulero@urv.cat

Assessment
Methodologies Competences Description Weight        
Oral tests
Test that will be carried out during the development of the seminar by the student. It will be evaluated the content and the format, as well as the knowledge and the defense capacity. 10%
Extended-answer tests
A4
A13
A15
 Tests that include open questions about a topic. The student has to develop, relate, organize and present the knowledge he has on the subject. The answer which give the student has to be extensive. 90%
Others  
 
Other comments and second exam session

In the second call it will be evaluated the entire content of the subject. The seminar mark is saved.

During the evaluation tests, mobile phones, tablets and other devices that are not expressly authorized by the test must be turned off and out of sight. The demonstratively fraudulent realization of any evaluative activity of any subject in both material and virtual and electronic support entails the student the failure mark of this evaluative activity. Regardless of this, given the seriousness of the facts, the center can propose the initiation of a disciplinary file, which will be initiated by resolution of the rector.

Sources of information

Basic

1. A Guide to Human Gene therapy (Roland W.Herzog, Sergei Zolotukhin). World Scientific, 2010

2. Gene therapy (Mauro Giacca). Springer, 2010

3. Gene therapy: Prospective Technology in Its Social Context (Jörg Niewöhner adn Cristof Tannert). Elsevier, 2006

4. Gene therapy: The use of DNA as a drug. (Gavin Brooks). Pharmaceutical Press, 2002
Complementary

Recommendations


Subjects that it is recommended to have taken before
MOLECULAR GENETICS/19204113
RECOMBINANT DNA TECHNOLOGY/19204117
(*)The teaching guide is the document in which the URV publishes the information about all its courses. It is a public document and cannot be modified. Only in exceptional cases can it be revised by the competent agent or duly revised so that it is in line with current legislation.