CRISPRMED26 WORKSHOP SUCESS

The workshop will start with an introduction to various genome-editing technologies highlighting their mechanisms, advantages, and limitations.

We will also discuss recent developments in genome-editing tools, focusing on those DSB-free and with demonstrated therapeutic potential, such as base- and prime editing. Next, we will cover criteria for selecting the appropriate genome-editing tool based on the targeted disease and present case studies of therapeutic applications.

We will emphasise that different diseases require distinct genome-editing strategies depending on the aim, such as gene replacement, gene restoration, or gene insertion.

The workshop will include a hands-on session with practical demonstrations and group activities to design genome-editing experiments. Finally, we will address ethical and safety considerations, emphasising regulatory guidelines and best practices.

The workshop will be highly interactive, with Q&A sessions throughout, pausing at key points for discussion.

This workshop will focus on navigating the current landscape of viral- and non-viral delivery methods for CRISPR gene-editing tools.

The session will provide the participants a broad and comparative introduction on the various delivery methods available for the different CRSPR gene-editing tools, their advantages and disadvantages.

Importantly, the workshop will try to achieve a comprehensive understanding on how to select the right delivery tool for a specific CRISPR gene editing in cells to achieve high efficacy, the so-called “CRISPR-Delivery-Cell, CDC” match-making procedure.

This workshop will focus on the side effects derived from the use of genome editors. The session will address the risks associated with the unspecific activity of CRISPR-based platforms, which can impact both the safety and efficacy of gene-editing approaches. Participants will gain a comprehensive understanding of the different types of potential genotoxic events and of the different methods available to monitor unintended genomic modifications, including those aligned with regulatory guidelines:

–        Off-target activity of genome editors

–        Genotoxicity: on- and off-target aberrations

–        Off-target evaluation methods

–        Mitigation strategies

–        Hands-on basic training on specificity prediction