Ateneo - UPO

This course will cover the main principles of cell engineering and explore novel therapies that activate the immune system, which play a key role in the treatment of hematological malignances. The course will begin with an overview of the diseases of interest, followed by a description of the methodologies and clinical efficacy of these innovative therapeutic approaches.

Teaching materials used during lessons (slides, etc) will be pivotal for exam preparation As an additional learning tool for consultation: Williams Hematology, 9e Author(s): Kenneth Kaushansky, Marshall A. Lichtman, Josef T. Prchal, Marcel M. Levi, Oliver W. Press, Linda J. Burns and Michael Caligiuri (available on UPO off campus library via AccessMedicine)

The aim of the course is to provide students with the knowledge required to understand the biological and clinical rationale underlying diseases for which cell engineering techniques are currently employed, and to acquire the fundamental principles of cell engineering relevant to a Medical Biotechnologist. The course also aims to provide a deeper understanding of the mechanisms of action of novel immunomodulatory drugs, such as bispecific antibodies. The course also aims to develop students’ ability to critically analyse and apply this knowledge, in accordance with Dublin Descriptor 3.

Background in molecular biology

A) Taught (frontal) classes with slide presentations available on the UPO DIR website (https://www.dir.uniupo.it/);
B) Group presentations by the students on topics agreed upon with the professor

Students with physical disabilities, Learning Disabilities or Special Education Needs can request specific services and tools via the , consulting the University webpage: https://www.uniupo.it/en/services/services- students-physical-or-learning-disabilities Students with disabilities, learning disabilities or special education needs, once they have contacted the University Staff, can refer to the tutor in charge of the course to define the examination modalities, concerning academic aspects.

Multiple-choice exam and open-ended questions

CAR-T Cell Therapy
Overview of diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and B-cell acute lymphoblastic leukaemia (B-ALL). Manufacturing of CAR-T cells. Clinical efficacy of CAR-T cell therapy in different lymphoma subtypes. Adverse events associated with CAR-T cell therapy. CAR-NK cell therapy. Mechanisms of resistance to CAR-based therapies.

Bispecific Antibodies.
Rationale, mechanism of action, toxicity, and mechanisms of resistance.

Introduction to Cell Therapy for Haemophilia, Thalassaemia, and Sickle Cell Disease
Overview of the coagulation cascade and the different types of haemophilia; structure and function of haemoglobin; overview of the different types of thalassaemia; pathophysiology of sickle cell disease; gene therapy for haemophilia; and gene therapy strategies for thalassaemia and sickle cell disease

Students will learn the basics of gene therapy as applied to lymphomas, hemophilia, and thalassemia. Students will also learn the fundamental principles of novel drugs that activate the immune system.