Novas Terapias Celulares e Moleculares

Objectives

The aim of the UC is to provide the student with an integrated vision of the potential of the novel Cellular and Molecular Therapies. The student should acquire deeper knowledge of the genetic, molecular basis for implementation of novel therapies for disease correction. Understand the biological mechanisms of repair versus regeneration. Understand the definition and concept of embryonic stem cell in comparison to somatic cells.

 The aim is to provide general knowledge and integrated concepts in stem cell biology at the molecular, cellular and animal levels. Display the properties, the potential and limitations of using pluripotent stemcells (embryonic or reprogrammed – such as iPS) and adult regenerative medicine.

Exogenous DNA delivery technologies for the correction of genetic defects. Strategies for correcting genetic defects for genome correction and / or protein production, use of viral and nonviral vectors.

General characterization

Code

121004

Credits

3

Responsible teacher

Professor Doutor José António Henriques de Conde Belo

Hours

Weekly - Available soon

Total - Available soon

Teaching language

Portuguese

Prerequisites

 

Bibliography

·         StemCells: From Mechanisms to Technologies” - By Michal K. Stachowiak, Emmanuel S. Tzanakakis – World Scientific Publishing Co, 2012.

·         Stem Cells and Cancer StemCells, Volume 2: Therapeutic applications in disease and injury” - by M.A. Hayat – Sringer, 2011.

·         Principles of Regenerative Medicine” - By Anthony Atala, Robert Lanza, James A. Thomson, Robert Nerem – Academic press, Elsevier, 2010.

·         Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition, Edited by Nancy Smyth Templeton.

·         Scientific articles and other support material.

Teaching method

Half of the classes will be related with syllabus teach ing, being available for students all slides used in the classes. There will also be an important component of presentation of research seminars delivered by field experts. A significant part of the teaching time will be devoted to the presentation and discussion of various scientific articles, provided by the teachers. The presentations of papers will be performed by the students.

Evaluation method

The evaluation will be done through group presentation / discussion of papers (75% of grade) and the individual presentation of one paper (25% of grade). Attendance of a minimum of 80% of the classes is mandatory.

Subject matter

-    Introduction;

-    Basic concepts in Stem Cells and Gene Therapy;

-    Understand the biological mechanisms of repair versus regeneration;

-    Gene Therapy: objectives, therapeutic strategies;

-    Vectors for gene therapy;

-    Examples of therapeutic s trategies under development, under clinical trials and in commercialization;

-    Stem Cell Biology. Stemness and pluripotency;

-    Differentiation strategies;

-    Animal models of disease;

-    Mesenchymal, hematopoie tic, fat-derived, umbilical cord stem cells;

-    Characteristics, plasticity and mechanisms of action;

-    Therapeutic applications;

-    Tissue-specific stem cells: Neural, muscle, pancreas, skin;

-    Identification and isolation. Importance of the niche;

-    Applications in animal models of disease;

-    iPSC and transdifferentiated cells;

-    Reprogramming principles, methods limitations and advantages;

-    Directed repro gramming (transdifferentiation) of somatic cells into cells with diferente Functions.

Programs

Programs where the course is taught: