Digital Workshop Day

Workshop A | 9:00am-12:00pm

RNA Editing: An Introduction

As RNA editing is set to overtake the likes of CRISPR and DNA editing, it could allow clinicians to make temporary fixes that eliminate mutations in proteins, halt their production or change the way that they work in specific organs and tissues. As excitement in the space grows, the need to understand the fundamental basics of RNA editing is critical in propelling this field forward.
Attend this workshop to discuss:

  • How to target CàU editing in disease?
  •  How to target AàI editing in disease?
  • Z-binding domain
    • Deaminase Domain
    • Interferon Pathway
  • How best to target edits?
    • Oligonucleotides to target endogenous Adar enzymes
    • Editing cassettes that supply both enzyme and targeting
    • Safety versus Delivery
  • Target editing to DNA or RNA?
    • in vivo or ex vivo
    • Durability and efficiency
  • How to decide whether editing is safe?
    • On target versus off-target
    • Adverse immune effects
    • Age and sex considerations

Workshop Leader

alan herbert insideoutbio

Alan Herbert
Founder & Head
of Discovery

Workshop Leader

Riccardo Pecori

Riccardo Pecori
Division of Immune Diversity
German Cancer Research Center

Workshop B | 1:00pm-2:00pm

Enabling the RNA Platform to be the Tool Box for Precision Medicine

RNA editing has the potential to be a powerful tool to study the function of specific genes and proteins important for development or disease. There are still several hurdles that have to be overcome, but patient-tailored therapies seem to be within reach.
Join this interactive workshop to:

  • Intro to ProQR, and the business - Developing an RNA editing precision medicine
  • Our Axiomer A-->I editing platform - How to develop a rational approach to efficient development of precision medicine
  • Our expanding interest in base editing; introduction to our Trident platform on pseudo uridylation
  • Developing highly predictive preclinical models to improve the probability of success of precision medicines

Workshop Leader

Gerard Platenburg

Gerard Platenberg
ProQR Therapeutics