Neurobiology

GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module I – Isabella Saggio Program 1. Technology The course will cover gene and cell therapy tools 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 aims to explore the most recent concepts regarding the genetic basis and potential therapies of diseases, including those affecting the nervous system, age-related disorders, and cancer. GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module II – Romina Burla Program Clinical experimentation The course includes an analysis of the scientific literature concerning the application of gene therapy approaches to major neurological diseases. Scientific papers covering both pre-clinical and clinical phases of gene therapy, developed for the treatment of genetic diseases in general and neurological disorders in particular, will be examined.

GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module I – Isabella Saggio Prerequisites Basic knowledge of genetics and molecular biology. GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module II – Romina Burla Prerequisites Basic knowledge of genetics and molecular biology. Knowledge of gene therapy tools acquired in Module I.

GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module I – Isabella Saggio Adopted 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 and Cell Therapy: Therapeutic Mechanisms and Strategies / ed. Nancy S. Templeton (2015) ISBN 978-0849387685 The Development of Human Gene Therapy / ed. Theodore Friedmann (1999) ISBN 978-0879695286 Gene Therapy: Principles and Applications / ed. Thomas Blankenstein (1999) ISBN 978-3764359720 Cancer Gene Therapy / eds. David T. Curiel, Joanne T. Douglas (2005) ISBN 978-1588292131 Concepts in Genetic Medicine / eds. Boro Dropulic, Barrie Carter (2008) ISBN 978-0470184585 Gene Therapy in Neurological Disorders (1st ed., 2018) ISBN 978-0128098134 The textbooks are available for consultation at the Library of the Department of Biology and Biotechnology “Charles Darwin.” GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module II – Romina Burla Adopted textbook 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.

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

GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module I – Isabella Saggio Assessment methods The exam will consist of the discussion of a research project, resulting from the laboratory and tutoring activities carried out during the course. The examination may be conducted orally or in written form with open-ended questions. GENE THERAPY AND NEUROSCIENCE 2025/2026 – Module II – Romina Burla Assessment methods The exam will consist of the discussion of a scientific paper, as presented and discussed during the course. The examination may be conducted orally or in written form with open-ended questions.

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

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