Degree course: 
Corso di Second cycle degree in BIOMEDICAL SCIENCES
Academic year when starting the degree: 
Academic year in which the course will be held: 
Course type: 
Supplementary compulsory subjects
First Semester
Standard lectures hours: 
Detail of lecture’s hours: 
Lesson (16 hours), Laboratory (24 hours)

to fully understand the topics addressed in the course, students should possess basic notions of molecular and cellular biology.

Final Examination: 

During the final oral exam the student must present a scientific paper focused on one of the topics of the course. The student must critically discuss the scientific question and the methods comparing this with the general topics of the course. The student’s comprehension and presentation of the specific paper will be evaluated together with a general overview of the topics with particular attention on the practical part (laboratory) of the course.

Voto Finale

RNA molecules have in the recent years become a novel area of interest in the development of experimental and therapeutic strategies against monogenetic disorders, cancer and infectious diseases. RNAs are unique in their capacity of interacting with three major groups of biological macromolecules: DNAs, RNAs, and proteins. Therefore, RNA therapeutics will expand the range of druggable targets including conventional proteins, and previously undrugged or undruggable transcripts and genes.
The course aims at providing an updated overview of the molecular and cellular mechanisms of current therapeutic/experimental approaches based on RNA drugs.
Students will acquire a comprehensive knowledge of the different types of RNA drugs such as antisense oligonucleotides (ASOs), RNA aptamers, small interfering RNAs, microRNAs, mRNAs, and the guide RNA based CRISPR/Cas9 system used for genome editing. Their mechanism of action will be analyzed together with the challenges concerning their stability, specificity, safety, and delivery.
The laboratory module will allow the students to develop practical skills and test some RNA based drugs in vitro in cell models. A fundamental part of the laboratory is to critically evaluate the experimental design and interpretation and to summarize and expose the obtained data to the classmates and the teacher taking into consideration also scientific literature.

Classes of RNA drugs: ASOs, RNA aptamers, small interfering RNAs, microRNAs, mRNAs, guide RNA (gRNAs);
Mechanisms of action: gene knockdown, splicing modulation, trans-splicing, gene editing, protein substitution;
Laboratory module:
Two laboratory experiences are included in the program of the course in which two different classes of RNA molecules will be introduced into cultured cells and the effect on specific phenotypes analyzed. The students will be divided into small groups of 2 persons/group to facilitate the “hands-on experience” as well as working in a team.
Experience #1 is based on testing splicing correction using a small RNA while experience #2 is based on the CRISPR/Cas9 system.

Safety and specificity concerns;
Delivery systems.


Frontal lectures are based on PowerPoint presentations. The students will be stimulated to participate in a direct dialogue with the teacher and classmates.
The laboratory module consists of 24 hours of laboratory practice during which the experimental design and approaches will be discussed with the students.