Knowledge and understanding: the student will have to show acquired knowledge on the techniques and objectives of gene therapy, cell therapy and tissue engineering, as well as on the state-of-the-art of existing clinical trials.

Applied knowledge and undestanding: the student will have to show the acquisition of specific skills and competence in the analysis of the experimental procedures used in pre-clinical and clinical studies of gene and cell therapy.

Making judgements: the student will have to show capacity to use the acquired knowledge to critically analyze the results obtained by these therapies.

Communication skills: the student will be stimulated to acquire a proper language and to expose technical and scientific concepts in a professional manner.

Learning skills: the student will have to show the ability to retrieve information on regenerative medicine in an independent manner through the access to scientific publication. This capacity will be fostered by the leaded discussion of papers recently published in the literature.

Knowledge of cellular and molecular biology

The course addresses the main topics relevant to gene therapy and regenerative medicine, with particular reference to the molecular mechanisms underlying age-related degenerative diseases, and innovative biological therapies (recombinant proteins, gene therapy, cell therapy) that aim to interfere with these mechanisms.
Topics are in line with the objectives listed in the 'articolo 2 del Regolamento Didattico del Corso di Studi'

Giacca - Terapia Genica. Springer

Watson, Molecular Biology of the gene

Additional material distributed during lessons

Molecular Biology of Aging.
Stem cells: definition and features
Adult stem cells. Bone marrow stem cells and clinical use.
Regenerative medicine. Stem cell plasticity.
Ethical considerations. Intellectual property.

Embryonic stem cells: derivation, ES cell culture, plasticity, differentiation and genetic manipulation.
Cloning. The developmental program is reversible. Dolly the sheep. Animal cloning. Telomeres. Human cloning. Ethical considerations. Therapeutic cloning.
Epigenetics (chromatin remodeling and DNA methylation during cloning).
iPS cells: the reprogramming of a somatic cell into a ES-like cell. The stemness genes. Direct transdifferentiation.
Cell therapy of cardiovascular diseases
Cell therapy of neurodegenerative diseases
Cell therapy of diabetes
Molecular biology of aging
Stem cells and tissue engineering

Introduction to gene therapy and history
Gene therapy clinical trials, Therapeutic genes
Gene therapy of hematopoietic stem cells
Gene therapy of muscular dystrophies
Gene therapy of hemophilia
Gene therapy of neurodegeneration
Cancer gene therapy
Gene therapy of eye disorders
Gene therapy of cardiovascular diseases
Gene editing for gene correction. Applications and ethics

Theoretical lessons with the help of electronic presentations, which will be made available to students on the Teams platform.

Students will have to fill a questionnaire with 3 open questions and 12 multiple choice questions, aimed at evaluating the acquired knowledge and the capacity to critically analyze the results of clinical trials. Correct answers can be: one, more than one, all or none and each answer will be scored up to 2 points.
Ongoing evaluation sessions will allow assessing the competences and knowledge acquired during the course. Any changes these indications, which may become necessary to ensure the application of safety protocols related to the COVID19 emergency, will be communicated on the Department's and Degree Course websites and Lecture course Moodle page.
The final evaluation will be expressed as a score (from 0 to 30 thirty), according to the following grid:
- 30 e 30 e lode: perfect knowledge and language; exceptional analytical skills; the student can perfectly apply the acquired knowledge to concrete cases.
-27-29: good knowledge, language and analytical skills; the student can apply the acquired knowledge to concrete cases.
24-26: good knowledge of main topics and appropriate language; the student can often apply the acquired knowledge to concrete cases.
21-23: the student is not very familiar with all topics, while having fundamental knowledge; the language is often appropriate and the student can sometimes apply the acquired knowledge to concrete cases.
18-20: minimal knowledge of main topics and technical language; minimal capacity of applying the acquired knowledge to concrete cases.
<18: the student is not enough familiar with the course topics. To pass the exam, the student has to show a sufficient knowledge on all topics. To have the highest evaluation, the student has to answer correctly to all questions.

This course touch on topics closely connected with one or more goals included in the UN Agenda 2030 for Sustainable Development.