The A-T Society is delighted to announce that it has made a grant to support the project led by Dr Timothy Yu at Harvard at Boston Children’s Hospital which is attempting to repair the mutation in a child with A-T using a technique based on anti-sense oligonucleotides (ASOs).
You may have heard about this through emails and posts from Brad Margus of the A-T Children’s Project, which commissioned the project. ASO are short sections of RNA (very similar to DNA) which can potentially bind to the site of certain types of mutation to prevent the mutation from affecting the production of the ATM protein. ASO therapies have recently been shown to be effective in treating a number of different conditions, including Huntington’s disease and spinal muscular atrophy. They are injected into the spino-cerebellar fluid, and from there are able to reach the brain.
ASOs need to be made individually for each different mutation. They are also currently only a potential treatment for a limited number of mutations. Nevertheless, if the treatment proves effective, and this is still a big ‘if’, it will be the first time that someone has been successfully treated for A-T. This of itself will be a huge step forward for research and it will potentially open the door to treating more people.
The project is making good progress and has already demonstrated that an ASO can cause cells from a child with AT to produce ATM in a laboratory dish. There is still a long way to go to show that it is effective and safe in the body, but this is a very promising start.
If you have any questions about this project please feel free to contact us.