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Meet a leading Rett researcher

 

 

Ralph Hector is a geneticist who is working hard in search of our cure. Andy recently caught up with Ralph to find out what he has been working on and to show you the impact your support is making. 

 

Andy: Ralph thanks for making time to catch up with us. I don't think I've ever asked how you came to be researching Rett Syndrome!

Ralph: Well, I have always been involved in genetic research, but not always working on projects very close to the clinic. I was lucky that just when I decided I wanted to change direction and work on genetic therapies an opportunity arose in Stuart Cobb’s lab. The projects were very exciting, so I came on board initially as a keen scientist, not knowing anyone personally who was affected by Rett. Since then I’ve met so many families at fundraisers, events and conferences that I now feel part of a Rett community, which is fantastically motivating for a researcher.

 

Andy:  It's great to know we haven't put you off! What do you see as the big opportunities in Rett research over the next few years? 

Ralph: The gene therapy field is very exciting now, and it’s amazing that we have a Rett syndrome clinical trial in the pipeline. In the past 18 months we’ve seen some examples of truly transformative gene therapy for other disorders. Although Rett is particularly challenging, I think replacing or fixing MECP2 is our best bet for finding a cure, and I think we’ll see significant advances in the next few years.

 

Andy:  That's great to hear - you mention some of the challenges of working on Rett, what particular problems are you currently trying to solve?

Ralph: Our main current goals is to finding a gene therapy that is as safe as possible. We know that too much MeCP2 in cells can be as detrimental as too little, so we’re putting a lot of effort into finding ways to better ‘regulate’ our gene therapies. There are many approaches we can take to do that, and we’re trying a lot of them!

 

Andy: There has recently been some discussion in the scientific literature about the balance between funding research for a cure and research for treatments of its symptoms. What are your thoughts about that?

Ralph: Like most things, there’s a balance, but there is reason to be optimistic that a cure is a realistic target. The word cure is often loaded with baggage, but I think families have a realistic, and optimistic, view of what that is for them. If ever there was a time to keep working towards a cure, it is now.

There have been significant advances in many areas of genetic therapy over the past 18 months, in gene therapy, RNA editing and X-chromosome reactivation. With so many different approaches targeting Rett at a fundamental level (by replacing or fixing MeCP2) now is not the time to take the foot off the gas with respect to developing a cure.

 

Andy: You recently moved to Edinburgh, which is where leading Rett scientist Sir Adrian Bird is also based; how will working more closely with Adrian help you to make progress?

Ralph: Having Adrian’s lab so close to us now makes it that much easier for our labs to meet up, discuss ideas and share data. Their research addresses so many of the important biological questions about Rett and MeCP2, and we’re collaborating with them to help develop these ideas into therapies at much earlier stage. We’re hopeful that this will allow us to progress promising therapies much quicker.

 

Andy:  It sounds like you have a lot to get on with! Before I let you go, what five words would you like to share with supporters of Reverse Rett?

Ralph: Believe in research for Rett.

 

Andy: Tea or coffee?

Ralph: Coffee!

 

Published: 3 May 2018 10:35