Knowledge and understanding. The student will prove knowledge on advanced therapies and on the main methods used for their development. The student will be familiar with the major advanced therapies that have reached the clinic, the diseases for which they are used, their pharmacological and biological properties and their mechanisms of action.

Capacity to apply knowledge and understanding. The student will prove independency in evaluating the technologies applied to a specific contest (drug development, formulation, production).

Making judgments. The student will be able to elaborate on the topics of the course in a critical and independent manner. The student will critically analyse the results already obtained and those that will reasonably be obtained in the near future by advanced therapies.

Communication skills. The student will master the scientific and medical language and will communicate the concepts discussed during the course in a professional manner.

Learning skills. The student will be able to acquire novel information on advanced therapies by retrieving the relevant scientific publications. This capacity will be stimulated by the guided discussion of recently published papers.

Knowledge of genetics and molecular biology, introduction to clinical practice

The Course provides information on the development and clinical application of Advanced Therapies. This definition includes gene therapies with viral and non viral vectors, therapies with small regulatory RNAs and mRNAs, therapies with cells, including stem cells, and therapies with tissues, including the use of biomaterials. The Course presents and discusses the current and future applications of gene therapy for hereditary disorders and of stem cell therapy for complex disorders (dementias, cardiovascular conditions, diabetes). Particular attention is paid to the implementation of new therapeutic modalities, including the use of small regulatory RNAs (siRNAs and microRNAs), engineered cells for the immunotherapy of cancer (T-CARs) and the CRISPR/Cas9 technology for the precise editing of the human genome

Giacca "Gene Therapy" Springer ed. Review articles suggested in the classes

• Introduction to advanced therapies. From insulin to genome editing. The revolution of bio(techno)logical drugs. Specific characteristics of biological drugs and advanced therapies.
• Gene therapy. Viral vectors (retroviruses and lentiviruses for immunodeficiencies and other blood diseases: adenovirus for cancer; AAV for disorders of muscle, eye and central nervous system). Antisense oligonucleotides and regulation of splicing. Small regulatory RNAs (siRNAs, shRNAs and microRNAs) for infectious and complex disorders. Aptamers for diseases of the eye.
• Cell therapy. Stem cells. Cell therapy of diseases of the cardiovascular and nervous systems, of the eye and of diabetes. Engineered T- lymphocytes (T-CARs) for the therapy of hematopoietic malignancies and solid cancers.
• Precise gene editing. CRISPR/Cas9 technology and its therapeutic applications.
• Precision medicine

In person traditional lessons with presentation of slides. All the material shown is available to the students on the Microsoft Teams platform. Lessons are recorded.

Slide decks used for lessons will be made available to students

Conversation. Outcome is binary (passed/not passed). The student will discuss topics in the advanced therapy area. To pass, the student must show familiarity with the main methods for the development of advanced biological therapies.

SDG3 Good health and well being. This teaching explores topics closely related to one or more of the goals of the United Nations 2030 Agenda for Sustainable Development.