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
    nucleotide
    • 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
InsideOutBio

Workshop B | 1:00pm-4:00pm

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:

  • How to develop a rational approach to efficient development of precision medicine
  • Developing an RNA editing precision medicine
  • What’s the regulatory perceptive on accelerating the development of precision medicines using RNA editing technologies?
  • Developing highly predictive preclinical models to improve the probability of success of precision medicines

Workshop Leader

ProQR_David Rodman

David Rodman
Executive Vice President
ProQR Therapeutics