Molecular Therapeutics and Tissue Engineering

Recommended Prerequisites

Molecular biology, physiopathology and studying models of diseases.

Teaching Methods

Master classes with description and illustration of the concepts using slides. Use of multimedia and laboratory part of filming done in FFUC. Formulation based learning. Presentation of scientific articles.

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 functions

Perform basic and: applied research in the biomedical field

 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 regulatory aspects

Introduction or replacement of genes

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

Non-viral vectors: Lipid- and polymer-based nanoparticles, exosomes.

Addition of therapeutic genes

Nucleic acid-based 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 - biomaterials and its applications

Stem Cells - Types of stem cells

Embryonic stem cells

Induced pluripotent stem cells

Neural stem cells

Mesenchymal stem cells

"In vitro" studying models.

Head Lecturer(s)

António José Ribeiro

Assessment Methods

Assessment
Other: 20.0%
Synthesis work: 40.0%
Exam: 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.

Costa, B., Boueri, B., Oliveira, C., Silveira, I., Ribeiro, A.J., 2022. Lipoplexes and polyplexes as nucleic acids delivery nanosystems: The current state and future considerations. Exp. Opin. Drug Deliv. 19, 577-594.

Delaunay, S., Helm, M., Frye, M., 2023. RNA modifications in physiology and disease: towards clinical applications. Nature Reviews Genetics.10.1038/s41576-023-00645-2

Nikom, D., Zheng, S., 2023. Alternative splicing in neurodegenerative disease and the promise of RNA therapies. Nature Reviews Neuroscience 24, 457-473.