HELP!!:Gene Therapy for Huntington's Disease

Dr David E Barton dbarton at
Wed Jul 26 08:24:11 EST 1995

In article <3v2hs2$1hq at>,
Brendon Chua  <avalon at> wrote:

>I'm currently doing a thesis on the recent dicovery of the gene segment 
>on chromosome 4 that causes Huntington's disease. I am in urgent need of 
>any information on the gene therapies and genetic treatments availible 
>for this disease if any.

As far as I am aware, there is no treatment available, and developing gene    
therapy is going to be a formiddable challenge, because the tissues involved  
as so inaccessable, and because the disease is dominant. That is to say that  
every cell in an affected individual already has a normal copy of the HD gene 
in it, so just getting a normal gene in is not going to work, like it would   
for an enzyme deficiency. Therapy is likely to be directed at knocking out the
mutant gene, but how do you knock out a gene with 40 repeats and leave the    
normal one, which might have 30 repeats, intact?

All suggestions welcomed!!

  | David Barton
  | National Centre for Medical Genetics
  | Our Lady's Hospital for Sick Children
  | Crumlin, Dublin 12, Ireland.
  | Tel +353 1 455 0515 Fax 455 8873

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