Molecular Therapeutics and Tissue Engineering

Recommended Prerequisites

All areas of life sciences (examples: Pharmaceutical Sciences, Medicine, Biochemistry or Biology).

Teaching Methods

Lectures: students actively engaged in the construction of knowledge, analysing diverse information in the context of Molecular Therapy and Tissue Engineering. Links between a professional context and exploited scientific concepts will thus be established.

Autonomous learning: students will be encouraged to, autonomously get actively involved in the construction and development of their knowledge, searching and analysing information and solving problems, proposed (or not) by the teacher.

Problem-solving class: Presentation of papers selected within the themes of the lectures.

Learning Outcomes

This course is primarily intended to confer knowledge about new products and therapeutic strategies with a molecular basis in the areas of Biotechnology, Genetic Engineering and Tissue Engineering.

The course will contribute to enable students at the end of their training to:

• Perform basic and applied research in the biomedical field

• Perform functions:

- in the selection, use and evaluation of new methods of molecular and cell therapy

- in the monitoring of clinical trials of new drugs and / or therapeutic strategies resulting from advanced technologies

- in the regulatory area of ​​advanced therapies and biotechnology medicines and implementation of new therapeutic strategies

The implementation of the objectives described will enable students to develop the following skills: organization and planning; manage information; problem solving; critical thinking; autonomous learning; oral and written communication; concern for quality.

Work Placement(s)

No

Syllabus

Gene therapy; definitions and enforcement aspects

Introduction or replacement of genes

Viral vectors: adenoassociated viral vectors, lentiviral vectors, retroviral vectors, adenoviral vectors.

Non-viral vectors

Addition of therapeutic genes

DNA vaccines

Modulation of gene expression

Antisense oligonucleotides, siRNA, and ribozymes ml RNA: chemical structure and molecular mechanisms of gene expression silencing

Repair gene mediated nucleic acids: Zinc Fingers Talen and CRISPR

Therapeutic cloning: cellular engineering for regeneration and tissue repair

Clinical application of gene therapy protocols; in vivo and ex vivo strategies

Tissue engineering - Introductory concepts

Stem Cells - Types of stem cells

Embryonic stem cells

Induced pluripotent stem cells

Neural stem cells

Mesenchymal stem cells

Use of biomaterials for tissue engineering.

Head Lecturer(s)

Luís Fernando Morgado Pereira de Almeida

Assessment Methods

Assessment
Clipping presentation: 20.0%
Exam: 40.0%
Presentation: 40.0%

Bibliography

• Strachan, T and Read, A, Human Molecular Genetics 3, Garland Science, Taylor and Francis, London, 2003.
• Templeton, N and Lasic, D, Gene therapy. Therapeutic mechanisms and strategies, Marcel Dekker, New York, 2000.
• Gregoriadis, G and McCormack, B, Targeting of Drugs. Strategies for Gene Constructs and Delivery, IOS Press, Amsterdam, 2000.
• Nobre RJ and de Almeida LP. Gene Therapy for Parkinson's and Alzheimer's Diseases: From the Bench to Clinical Trials. Curr Pharm Des. 2011;17(31):3434-45.
• Cavazzana-Calvo, M, Thrasher RA and Mavillio, F (2004) – The future of gene therapy: balancing the risks and benefits of clinical trials. Nature. 427, 779-781.
• Nathwani AC, Davidoff AM and Linch DC (2005) A review of gene therapy for haematological disorders. Br J Haematol., 128, 3-17.

Clarke PA, te Poele R and Workman P (2004) Gene expression microarray technologies in the development of new therapeutic agents. Eur J Cancer. 40, 2560-91.