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• Breakthrough /Gene Therapy
• Posted: 2008-04-28 06:56:37 By mohammed s
• Hi all
  I got an exciting email few minutes ago from the Foundation Fighting Blindness .I think you all got it as well.Please raise the topic here and discuss about this breakthrough of gene theraphy.When are we going to get this treatment?
  I especially look forward to hear from Darran .
  Darran you there?? knock !! knock!!!
  Everyone stay fine
  
  Salam

• Re: Breakthrough /Gene Therapy
• Posted: 2008-04-28 11:23:52 By Bryan C
• Yes, I just received this very exciting news as well.
  
  I am not too familiar with the pace of clinical trials beyond knowing that they move slowly to assure their accuracy and safety. Clearly, if this trial is already being conducted in people it is much more advanced.
  
  Does anyone know what happens when a human trial gets positive results in terms of widening the number of participants? Will there be a long holding pattern with the current group to assure there are no adverse effects?
  
  If anyone knows the process that can share, I would love to hear.
  
  Thanks

• Re: Re: Breakthrough /Gene Therapy
• Posted: 2008-04-30 14:45:03 By Carol C
• This IS exciting, but I think some of the limits will be that each gene will have to have it's own designed injection, and there are so many different gene mutations involved in RP. It might be a good idea for as many people as possible to get their mutation identified, if only to be identifiable if research becomes available for your mutation. I beleive the article said that there are 12 genes identified causing LCA, and this was designed for only one. Carol

• Re: Breakthrough /Gene Therapy
• Posted: 2008-04-28 18:00:14 By Darran Z
• Well, here is the official publication at the NEJM:
  
  http://content.nejm.org/cgi/content/full/NEJMoa0802268
  
  After reading through this, it's not as far along as the media tried to hype it as. It's a step in the right direction. This is also not the first human gene trial to ever take place. The first one went bad and the person died. It has taken them 10 years to get to this stage, albeit a small step but a very important step. Gene therapy is still years/decades away from a reality.
  
  Darran