Thursday 22 January 2015

'Designer babies' and cystic fibrosis: Where's the connection?

Our Director of Research and Care, Dr Janet Allen, takes us through the recent discussion around 'designer babies' and where cystic fibrosis fits in.

The recent media focus on ‘designer babies’ has challenged thinking about what the future could hold; using tools to correct mutations in genes such as those that cause cystic fibrosis. 

It is important to understand that the correction of mutations (also known as genetic editing) can be done in the laboratory today using CRISPR technology. In fact the Trust is already funding two programmes in this area for cystic fibrosis. At the same time, a small biotechnology company has been pioneering a slightly different approach of repair called RNA editing and is about to start on very early clinical studies. 

Gene or RNA editing are both forms of gene therapy, although different from conventional gene therapy which we have been funding through the Gene Therapy Consortium. Conventional gene therapy inserts the corrected gene into the airway cells; in the editing approach, the mutation in the person’s own gene or RNA is corrected. The big question is whether it is possible to transfer the laboratory successes and correct CF-causing mutations in people’s lungs.

These are early days and already people are thinking about the next, next generation of gene therapy, combining genetic editing with stem cells to overcome constraints on delivering the corrected gene. It is possible to take cells from a person and, in the laboratory, convert them into stem cells (called iPS) specific to that individual. The CF-causing mutation can then be edited in the laboratory and the stem cells converted back to lung cells. A Trust-funded project is just about ready to start and we should know by the end of this year whether it has been successful in the laboratory.

So what does this all mean for the designer baby debate? Well the technology to edit out the CF causing mutation is exactly the same for designer babies as the next generation and next, next generation gene therapy approaches already taking place. 

For designer babies, this editing would need to be completed in the embryo and this is where the ethical considerations become the overriding issue and it is good that we’ve been challenged to start the debate, well before any of this advances. The debate gets tricky because the same technology that could correct for conditions such as cystic fibrosis could be used for non-health issues, such as hair or skin colour that a parent deems less desirable.

In the UK, we have an excellent framework to control research activity and ensure topics like these are well-thought through and all ethical considerations given time. Legally, this area is tightly controlled by the Human Fertilisation and Embryology Act 1990. Pioneers in the UK such as Dame Mary Warnock developed the thinking and ethics that allowed IVF to start and the recent ‘three parent’ programmes for some muscular dystrophies. In many respects, through the formation of the Human Fertilisation and Embryology Authority, these pioneers have provided the framework to safely explore challenging scientific advances such as designer babies and for the UK to lead the debate and provide thought-leadership in this area. It is good to hear also that the highly respected Nuffield Council on Bioethics is also considering the matter.  

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