I've spent the last three weeks working on a major article about a new gene silencing technique. It's complicated and it took me awhile to understand the two articles I'm covering plus some related articles I also cite. I've written about the new approach as well as an overview of where we stand with gene silencing progress in general. I hope you all will read it and I'll try to answer any questions you have. I think this is incredibly exciting.

[www.hdsa.org]

With these new techniques...would earlier treatment effectively short circuit the hd symptoms? Can it do any good for people who are now diagnosed as mild to moderate hd? How do you volunteer to be a guinea pig?

I have to say I am so amazed and hopeful by the new treatments that are coming. Also, I am extremely grateful to you, Marsha, for writing these things here.

Oh my god Marsha! Thank you VERY much, for your article, and your thorough info on each of the methods, and how this works, this looks so hopeful through several avenues, thank you!

Marsha...hmmm, sooooo, if they are able to refine the process for just fixing the bad cag, and lowering it into a normal range,..hmmm, if a baby is conceived, after the hd spouse has had the treatment, would hd be ended in that family????

For FevreDream:

If treatment in people parallels the results in HD mice, gene silencing, even later in the disease process, will partially reverse the disease. So yes, this treatment will certainly be for people with mild to moderate HD symptoms. Also, don't forget that restorative treatments are on the way, too. Researchers are now able to take skin cells from HD patients, correct the gene defect, treat the cells to induce them to return to their stem cell days, then turn them into medium spiny neurons. If treatment in people parallels that of treatment in mice, the cells will persist and be able to communicate with other cells and form circuits. See these articles:

[www.hdsa.org]
[www.hdsa.org]
[www.hdsa.org]

Barb: this method of gene silencing doesn't eliminate the bag CAGs, it just silences the HD gene, plus it goes into the brain, so there wouldn't be any effect on reproduction.

This is really exciting and hopeful news! But how long do you think it will be until these therapies are available to the public?

This is fantastic news, Marsha! Thank you so much for everything you do!!

Patty

Thanks Marsha for all that you do. It helps keep the hope of a cure strong in our hearts. You and Steve do an amazing thing here on this forum, it continues to be a lifeline for me and many other families out there.

Carla

Great news

I have just come home from teh Uk conefrence and lots of real hope coming from the gene silencing .

Wonderful news! One technique hinted at trials within 18 months. How can I find more information?
Thanks for all of your work to keep the community informed!!!

That's all the information I can find. I can answer questions if you'd like about the techniques. You can keep checking the website at Isis Pharmaceuticals and at Lundbeck and Alnylam for future announcements about trials.

Thank you Marsha, that is a great News for all of us.