Neurobiology

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Programme 1. Technology The course will cover the main tools of gene and cell therapy for the treatment of genetic and acquired diseases, including additive gene therapy using viral and non-viral systems, RNA interference, genome editing (CRISPR systems), and tissue engineering based on the use of progenitor and induced pluripotent stem cells (iPSCs). 2. Disease and experimentation The course will explore the latest concepts on the genetic basis and potential therapies for diseases, including those affecting the nervous system, conditions related to ageing processes, and cancer. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Programme 1. Basic elements of gene therapy for neurological diseases. 2. Case studies of gene therapy in neurological tumours. 3. Case studies of gene therapy in genetic and acquired diseases.

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Prerequisites Basic knowledge of genetics and molecular biology. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Prerequisites Basic knowledge of genetics and molecular biology.

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Textbooks Stem Cells: Biology and Engineering – Puc Van Pham (Springer, 2018) ISBN 978-3319774817 Cell and Gene Therapies (Advances and Controversies in Hematopoietic Transplantation and Cell Therapy) (1st ed., 2019) ISBN 978-3319543673 Gene Therapy in Neurological Disorders (1st ed., 2018) ISBN 978-0128098134 The course also includes the integration of these texts with recent scientific review and research articles. Books and articles are available for consultation at the Library of the Department of Biology and Biotechnology “Charles Darwin.” GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Textbooks Gene Therapy in Neurological Disorders – 2018, Editors: Mingjie Li and B. Joy Snider, Academic Press. ISBN: 978-0-12-809813-4 – DOI: 10.1016/C2015-0-04781-5 The course also includes the integration of this textbook with recent scientific review and research articles. Books and articles are available for consultation at the Library of the Department of Biology and Biotechnology “Charles Darwin.”

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Attendance Not mandatory. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Attendance Not mandatory.

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Assessment Methods The examination will consist of the discussion of a research project developed during the laboratory and tutoring activities held throughout the course. The final assessment will be conducted as an oral or written exam with open-ended questions. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Assessment Methods The examination will consist of the discussion of a scientific article. The final assessment will be conducted as an oral or written exam with open-ended questions.

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Reference Bibliography The bibliography used for the course is available and regularly updated on the e-learning platform in the course’s dedicated page. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Reference Bibliography The complete bibliography is available on the e-learning platform in the course’s dedicated page.

GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module I – Isabella Saggio Teaching Methods Lectures and laboratory activities accompanied by tutoring. GENE THERAPY AND NEUROSCIENCES 2025/2026 – Module II – Romina Burla Teaching Methods Lectures.