Ateneo - UPO

the principle of Cell and Gene Therapy
Stem cells
Gene transfer by viral and non viral vectors

For italians: Mauro Giacca, Terapia genica, Springer.
Animated gene therapy course, Utah University, USA

Journals:
Human Gene Therapy
Gene Therapy
Cancer Gene Therapy
Journal of Gene Medicine
Molecular Therapy
Nature
Science

Databases:
NCBI - MEDLINE, GenBank, Genomi, ecc.
Genetic Modification Clinical Research Information System (GeMCRIS)
Clinicaltrials USA
Wiley clinical trial database

The students should be come able to discuss cell and gene therapy published articles and fundamental vectorology used in gene therapy in preclinical studies and clinical trials.

Knowledge of Cell and Molecular biology from previous courses

Power point presentation and PDF articles

Group seminar prepared by the students on a chosen topic.

Students with physical disabilities, Learning Disabilities or Special Education Needs can request
specific services and tools via the Staff Sviluppo e Coordinamento Carriere e Servizi alle Studentesse

e agli Studenti, 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.

Written test on the topics of the program, with multiple choice questions and open questions. Moreover the students in groups they have to present a poster and an article with a commentary and a seminar discussion on a cell and gene therapy paper chosen by each group

Stem cells, cellular reprogramming and use of these cells in cell therapy protocols in preclinical and potential applications in the clinic.
Isolation, in vitro expansion and preparation of cells for experimental cell therapy.
Vectors used for gene transfer: viral vectors and non-viral. Origin, construction and modification.
Modulation of Expression cassettes: Constitutive and regulated expression. transcriptional or post-transcriptional expression regulation Immune response to vectors and transgenes used in gene transfer.
Concepts of ex vivo and in vivo gene therapy.
Clinical trials using cell and gene therapy strategies of target diseases.

Capacity to read and comment a scientific paper on cell and gene therapy