Gene therapy chondrotinase

[Adrian]

Chondroitinase gene therapy - not much detail in this and it is not near clinical trials in humans yet but it may be good in the future:

https://www.bbc.co.uk/news/health-44484901

[niallel]

Details on the project are here - https://www.spinal-research.org/chase-it

[niallel]

The was a segment on sky news today about it, but I can't find a link to the video. They did an article with the same info here:
https://news.sky.com/story/hope-for-...erapy-11404970

This was the first step though using an experimental lentiviral gene vector that has not been used clinically.

The next step is to use a Adeno-associated viral (AAV) vector that has been used in other medicines on the market. The thought is that by using that it will be easier for the regulators to allow it.

Professor Joost Verhaagen from the Netherlands Institute for Neuroscience is working on creating the AAV now.

What is most exciting is that ISRT are working to take this to the regulators so it can be used in clinical trials.

[Sythellri]

I’m excited!

[ineedmyelin]

The was a segment on sky news today about it, but I can't find a link to the video. They did an article with the same info here:
https://news.sky.com/story/hope-for-...erapy-11404970

This was the first step though using an experimental lentiviral gene vector that has not been used clinically.

The next step is to use a Adeno-associated viral (AAV) vector that has been used in other medicines on the market. The thought is that by using that it will be easier for the regulators to allow it.

Professor Joost Verhaagen from the Netherlands Institute for Neuroscience is working on creating the AAV now.

What is most exciting is that ISRT are working to take this to the regulators so it can be used in clinical trials.

https://www.youtube.com/watch?v=dCyIiDcn9WU

[Jade2]

Super cool! I always thought it was gonna be gene therapy!

[GRAMMY]

The full publication link in the Brain journal and Dr. Liz Bradbury video is HERE.

They've actually been working on the leaky vector for several years...

[niallel]

They've actually been working on the leaky vector for several years...

The AAV Vector work thats in progress hopes to improve on the lentiviral gene vector so they can stop any leaks. To me leaky doesn't describe the problem well, its more they can't turn every last bit off, but then again I've no idea about science. So quoting what I've been told:

"Using molecular biological techniques they will engineer a regulatable chondroitinase carrying AAV vector. In the first instance, the regulatable AAV vector will carry the gene for luciferase (a protein from the Firefly that gives off light when active), allowing the researchers a way of rapidly quantifying the levels of expression in vivo. Luciferase expression data obtained during four ‘on-off’ cycles will provide information about the inducibility and stability of doxycycline-regulated luciferase expression over time and about the leaky expression. Once regulation of luciferase is optimized in the AAV vector system, the luciferase gene will be replaced with the chondroitinase gene. This inducible chondroitinase AAV vector will then be tested by Professor Bradbury in a rat spinal cord contusion injury model."

If the chase bit doesn't work, at least we will all be able to glow up at night. It will save me crashing my wheelchair when I can't find the light switch :-)

[GRAMMY]

Originally when the large Chase team was formed, Elizabeth Muir was tasked with the job. That's been quite a few years ago. Nobody has yet been successful in getting the gene vector to shut totally off (leaky) with the use of DREADD. Perhaps Joost will get it mastered.

September 2012

https://spinalcordresearchandadvocac...cord-injuries/

I put up a post about the team and project on Nov 8th, 2012

https://spinalcordresearchandadvocac...hondroitinase/