June 18, 2025 – June 20, 2025
WEDNESDAY 18 JUNE 14:00-17:30
Welcome and Introduction
14.00 – 14.30
- Welcome from the GeneHumdi COST action and PhD School in Molecular and Regenerative Medicine
- Overview and Objectives of the workshop
(A. Cavazza, L. De Rosa, A. Recchia )
Session 1: Genome Engineering for Human Therapies
14:30 – 15:30 Lecture: Gene Editing in a Nutshell: dusting off the DNA repair pathways
15:30 – 15:40 Interactive Q&A with Mentimeter
15:40-16:00 Coffee break
Session 2: Genome Engineering Tools
16:00 – 16:30 Lecture: From Gen X to Gen Z: ZFN, TALEN and CRISPR-Cas (C. Mussolino)
16:30 – 17:00 Lecture: Epigenome editing (C. Mussolino)
17:00 – 17:20 Lecture: Base and Prime Editing (A. Carusillo)
17:20 – 17:30 Interactive Q&A with Mentimeter
THURSDAY 19 JUNE 9:30-17:30
Session 3: Delivery of genome editing components
09:30 – 10:30 Lecture: Delivery of engineering tools and donor DNA templates for HDR (A. Cavazza)
10:30 – 10:40 Interactive Q&A with Mentimeter
10:40-11:00 Coffee break
Session 4: Safety & Therapy design (Giando & Antonio)
11:00 – 11:45 Lecture Assessing safety for genome editing (G. Turchiano)
11:45 – 13:00 Practical session Intro “design and defend your safe and efficient gene therapy strategy: one disease, many tools”
13:00-14:00 Lunch
Session 4b
14:00 – 16:00 Practical session Hands on “design and defend your safe and efficient gene therapy strategy: one disease, many tools”
16:00-16:15 Coffee break
Session 4c
16:20-17:30 Discussion of gene editing approaches proposed by the working groups
FRIDAY 20 JUNE 9:30-13:00
Session 5: Clinical Application of Gene Editing
09:30 – 10:45 Lecture From bench to bedside: gene editing for clinical applications pt.1 (A. Cavazza, L. De Rosa, A. Recchia, C. Mussolino, G. Turchiano, A. Carusillo)
10:45-11:10 Coffee break
11:10-12:45: Lecture From bench to bedside: gene editing for clinical applications pt.2 (A. Cavazza, L. De Rosa, A. Recchia, C. Mussolino, G. Turchiano, A. Carusillo)
12:45-13:00 Closing Remarks
Details of hands-on sessions
Trainees are asked to bring along their own laptop for practical sessions and case studies.
Session 4
Practical session: Trainers will illustrate software for the design of gRNAs, HDR template, base editors, epigenome editors and to assess the safety.
Case study “One disease, many tools”: trainees will be split into working groups. Each working group will propose a custom approach to tackle a given disease taking into account potential efficacy and safety . Groups will prepare a short presentation and will present their proposed strategy.
