Anyone in the BHF available to answer questions on "£30m for scientists to rewrite DNA to cure killer heart diseases"

[Curryandchips]

How will it work?

Will it be suitable for older patients?

Will the heart be remodeled?

What are the dangers

How do you apply to be a guinea pig?

Who is going to keep us informed of progress?

[RufusScamp]

Good questions. I hope we get some answers. It might be good to enrol older patients on the grounds they have less to lose. Difficult, innit.

[Milkfairy Heart Star in reply to RufusScamp]

Here is some information from the BHF about Cure Heart

bhf.org.uk/cureheart?gclsrc...

[RufusScamp in reply to Milkfairy]

Thanks for that.

[Curryandchips in reply to RufusScamp]

I'm confused how they can overwrite established DNA and yet that is what i think is being suggested. I have HCA and would love to be part of the trials.

[HM24]

After reading the article, I wondered if anyone knows if it will only be applicable to those with a known HCM faulty gene. I have hereditory HCM, but the gene has not yet been identified?

[Steve_G]

The BHF have a nice article about this funding for gene therapy for inherited cardiomyopathy bhf.org.uk/what-we-do/news-...

The hope is that they can start trials/treatment within the next 5 years. The technology that will be used is based on CASPR-Cas9 gene editing to rewrite the wonky genetic code in the heart muscle cells that causes the problem. The condition will effectively be reversed by correcting the DNA genetic code. The challenge is developing a way to correct the DNA in all the affected heart muscle cells of a working heart in a body. The technology to do this in a test tube has been nailed but doing it in vivo, in a whole body, is still experimental. Both viral and non-viral vectors are being developed as a means of getting the treatment into the cells. This is likely to be one of the main challenges.

Over 10 years ago I had the privilege of working on the very first gene therapy to be approved but I’m in awe of the advances that have been made since then.

[Curryandchips in reply to Steve_G]

That's truly interesting. Can you explain how, physically, the heart changes after treatment? What happens to all the thickened the muscle?

[Steve_G]

Agreed, it’s fascinating and fantastic. Not that long ago it would have been science fiction but now it’s becoming reality. I’m no cardiovascular expert so can only provide assumptions based on my understanding and answer in general terms.

DNA codes the proteins that provide both cellular structure and the metabolic/catabolic function of the cell. If there’s a mis code then the structure and conformation of the protein expressed are affected. This means that cell structure are abnormal and/or enzymes don’t behave the way they should all of which leads to cardiomyopathy. The clever people at the BHF seem to have worked out which genes are involved and how both structure and enzymatic function are impacted. By replacing the faulty sections of the gene they will get the muscle cells to both look and function normally. The extent to which the heart structure is normalised is likely to be dependent on the proportion of the heart muscle cells that can be reached by the treatment but it’s likely that you only have to reach a proportion to gain the clinical benefit and significantly reduce the risk of death.

This definitely one to follow