Breakthroughs treating cystic fibrosis

[jreelawg]

http://www.breakthroughdigest.com/lung-diseases/gene-therapy-treatment-for-cystic-fibrosis-sufferers-in-the-works/

I have a friend who has cystic fibrosis, and she is already 24, with a life expectancy of about 30. Seams that with the ban lifted on stem cell research, and the breakthroughs in gene therapy, she just may live long enough to be cured.

Anyone have any knowledge of this type of thing? I am trying to find out who/where the leading researchers/ cutting edge innovation is happening, and how if possible to get in line, or possible sign up to be a test subject for something promising.

 

[Monique]

You should also read this article:
http://www.telegraph.co.uk/health/healthnews/4639764/Cystic-fibrosis-sufferers-offered-hope-by-gene-therapy-treatment.html

The researchers now hope to test the newly created virus in the first ever animal models of the disease, pigs bred last year, and then in humans.

They also believe that it could be modified to use against other diseases, including Alzheimer's.

A spokesman for the UK Cystic Fibrosis Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, said: “The techniques used in this research are innovative, but more work is needed before this could be translated into clinical benefit for people with CF.

The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene.

Although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient.”

Thus so far it has only been successfully studied in lung samples in the lab. There is still a lot of work to be done to test whether it work in a clinical setting and whether it is a safe treatment.

If you are really interested in entering the human trials, you could try and contact the researchers and ask whether that would be possible.

 

[Andy Resnick]

There's a couple of people here specifically trying gene therapy approaches to rescue the CF lung. There are major physiological barriers to transfecting cells in vivo (the mucus layer, the immune response, cell turnover, for example), and the only time (IIRC) this therapy was tried, the patient (18 year old) died.

There will probably be a flood of proposals in response to the US change in government policy, so it's too soon to tell what may happen. Stay tuned!

 

[ethan01]

Hi jreelawg, Cystic fibrosis is a rare genetic disease, and the best way to understand it fully is to do some research. Go and read this http://www.childwiki.com/index.php/Cystic_fibrosis" article for a detailed explanation.