The advances in genome editing can be traced back to quiet beginnings in the 1990s, but the current remarkable surge is largely because of the introduction of the CRISPR-CAS9, a genome-editing tool that can used to make changes in sections of the DNA sequence, in 2012. Recent scientific advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health.

The central question of this visionary seminar isn’t whether or not we will use human genome editing, but in which way and for which applications we will use it.

Programme

Abstracts

Eric Legius - Mutational mechanisms in human disease and genome editing potential

Human genetic diseases are characterized by many potential mutational mechanisms. Some mechanisms affect many hundreds of protein coding genes at once and other mechanisms are limited to a single gene such as simple changes of one letter of the genetic code into another letter resulting in a loss of function or a gain of function in the resulting protein. More complex mutational mechanisms are known and many mechanisms are not amenable to the current possibilities of genome editing.

Adrian Liston -CrispR: where are we at, and where are we going?

Just why is CrispR so revolutionary for gene therapy? In this talk, I will discuss what CrispR is, why it is so versatile and the different uses to which it is currently being put. For several classes of disease, the technology needed is now available, and just requires clinical optimisation. For other types of disease, new modifications of the CrispR system are still needed. I will end by discussing the potential future of CrispR in medical genome editing.

Hilde Van Esch - To CrispR or not to CrispR, biological pro and con’s of human genome editing

The recent discovery of the CRIPSR/Cas system has allowed for great improvement in the tools to modify genes and genomes. This has led to the development of many new animal models for research, but also enables editing specific target genes in human somatic cells, in germline cells, but also in a developing human embryo. This has raised many scientific issues and questions that we will discuss during this presentation.

Kris Dierickx - Ethical frameworks and social challenges of genome editing

The recent technologic discoveries in Human Genome Editing make us on the one hand dream of a world where curing genetic disorders is no longer a distand utopia. On the other hand, there are social and ethical questions in these developments: for which forms of ‘editing’ shall we choose as a society? Will it all be affordable and more accessible than the existing alternatives? What is the impact of such interventions on the following generations? And finally, can society and legislation keep up the pace of this rapid scientific progress?

Pieter Peeters and Araz Raoof - Genome editing at Janssen Pharmaceutica: towards disease interception and cure

In the first part of our talk we will address the application of recent advance in genome editing in pharmaceutical R&D. We will then switch to an overview of the biotech landscape in Genome Editing and the challenges in turning genome editing into a therapeutic application. Then we will talk about how we are driving towards cures for certain diseases of high medical need using genome editing to round up with our perspective on the future of this exciting technology.