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Is gene therapy for sporadic ALS on the horizon? Where does this stand?

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    Is gene therapy for sporadic ALS on the horizon? Where does this stand?

    Until recently I thought that gene therapy was only going to be available in the near(ish) future for those with familiar ALS, or a handful of causative mutuations such as in SOD1. However, I read an article today, reporting on a study that seems to challenge this view: Mutations in 22 Genes Linked to Sporadic ALS in Large Group Study ( It claims that 22 mutated genes have recently been discovered linked with sporadic ALS, and more intriguing that roughly 50% of people with sporadic ALS have 17 of these genes, which gives a 99% chance of developing the disease. I am a layman, and read this in a hurry, but to my eyes this suggests that for at least 50% of sporadic ALS patients, their disease is genetically determined, and it is possible that correcting these genes would slow down or arrest the disease. This study was only published recently and so it is difficult for a layperson to discern its significance - is this groundbreaking? Is there a real chance this could lead to gene therapies for sporadic ALS patients, and relatively soon? Thank you.

    Hello. I too believe that the key to treat ALS lies in the genetic makeup of each individual. The human body is a very complex organism. I am usually an optimist but I don’t think it’s realistic to expect a gene based treatment for ALS within the next 10 years. Research for genetic treatment is only at the very beginning and as you know, funding is and has always been a challenge.



      Hi all,

      We just wanted to share a response from our Research team.

      Unfortunately, we have just been informed that the paper you are discussing has some methodological issues, meaning that the results from this paper are potentially incorrect. We are currently in discussions with people involved with this, including someone who reviewed the original paper before it was published. We will make sure to update the community with exactly what happened and what went wrong once we have further information.

      Many thanks,

      Forum Admin
      Our working hours are Monday to Friday 8:30am until 5pm


        Last Wednesday Everything ALS Zoom Meeting had Dr. Michael Snyder from Standford Univ. on. He is a genius in DNA research and development. The 1 Hour Zoom should be posted soon on YouTube here.

        He says that his costs for full genome sequencing cost about $500US each. His team published a paper recently on this and he explained that they have identified around 600 genes associated with ALS/MND.