Tiny DNA Molecule May Help Development of Myelin Repair Therapies, Study Suggests

Tiny DNA Molecule May Help Development of Myelin Repair Therapies, Study Suggests

A shortened DNA molecule showed an increased ability to bind myelin in human cells, and may boost the development of remyelination approaches for multiple sclerosis (MS) treatment, according to a study.

The study, “Optimization of a 40-mer Antimyelin DNA Aptamer Identifies a 20-mer with Enhanced Properties for Potential Multiple Sclerosis Therapy,” was published in the journal Nucleic Acid Therapeutics.

Despite the availability of more treatments for MS, scientists are investigating potential approaches to restore the protective layer of nerve fibers, called myelin, which is progressively damaged in MS patients.

In this study, a team at Mayo Clinic College of Medicine and Science showed that a myelin-binding DNA aptamer — a small DNA molecule — known as LJM-3064 promoted remyelination in a mouse model of MS, and is rapidly distributed in the central nervous system (brain and spinal cord) as well as in other tissues of mice.

Compared with antibody-based approaches to induce remyelination, DNA aptamers are smaller, more stable, and easier and cheaper to produce. They also provide high target specificity and carry a low risk for immunogenicity, which refers to triggering an immune response.

The scientists shortened the original LJM-3064 from 40 to 20 nucleotides (the building blocks of DNA), hypothesizing that a specific portion of LJM-3064 was sufficient to bind myelin. A variant of this smaller molecule, called LJM-5708, showed increased myelin-binding ability.

LJM-5708 also retained the structure of the original 40-nucleotide compound. Importantly, the myelin-binding properties of LJM-5708 were preserved when the molecule was bound to a bacterial protein called streptavidin, which is key for improved body distribution and remyelination induced by aptamers in mice.

“These studies lay the framework for future testing of optimized remyelinating aptamer formulations in vivo,” the researchers wrote.

According to the team, the “optimized 20-nucleotide DNA aptamer LJM-5708 is thus a strong candidate for further preclinical testing, including … remyelination assay in vivo.” In particular, LJM-5708’s ability to specifically bind human cells makes it “a lead molecule for further investigation.”

‘The authors highlight the value of aptamer refinement of a therapeutic for multiple sclerosis treatment, and present a novel application for cell imaging,” Graham C. Parker, PhD, the journal’s executive editor, said in a press release.

28 comments

    • Dale Degraffenreid says:

      Absolutely there is a definite need for more PPMS market availability. Meanwhile, my daughter continues to battle PPMS that is killing her. Watching that is the worst thing to happen to any human being not to mention the pain she is suffering 24/7.

      • Cindy says:

        Try Charlotte’s Web Hemp cream. I get awful pains in my legs at night. I put this cream on my legs and the pain stops.

  1. Ed says:

    Repairing the myelin sheath does not repair the existing nerve damage. As someone with PPMS, I don’t understand why they keep throwing money down this rabbit hole.

  2. Harry says:

    Ya good luck on FDA approval. Anything that would cut into their pharmaceutical bonuses isn’t gonna happen soon or quickly. Stem cell treatment and many remyelination products are in the so called pipeline but it’ll cut into the pharmaceutical companies profits if they actually fix us instead of patching us. Its a shame. So many positive trial outcomes yet no approval. Especially on stem cell. Greed drives everything including our ability of being treated.

  3. Jeff Gregory says:

    DNA Aptamer LJM3064 shortened from 40 mer (nucleotides) to 20 mer (nucleotides) DNA Aptamer LJM5708 will bind myelin in human cells boosting the development of remyelination. Using myelin-binding DNA Aptamers to promote remyelination is unique but not much of any step in the right direction. Re programming the Lingo-1 negative regulatory protein to perform properly is the path. Begin the journey.

    • Christopher says:

      No, it is not. What you wrote makes no sense. The problem with remyelination is destruction of glial cells (specifically oligodendrocytes), not a signalling protein. There is no journey… just a long slog through mountainous amounts of experimentation.

  4. Helene m Patterson says:

    I wonder could those of us who have MS start a protest to bring the stem cells to the front. I wish i knew how I could get something like this going. We are the ones suffering. I agree that the pharmaceutical are to blame for cures. I makes me so mad, people who don’t have a illness don’t understand.

  5. Ken says:

    Dr. Terry Wahl, Take a look at what she is doing. Look at her TedTalk on Youtube. Her approach is working for her, and I doubt the cost of doing what she is doing, costs more than other health care approaches. Within months she was out of her chair walking again, and continues to improve.
    Ken

    • Michellr says:

      Dr Terry Wahl has never provided any proof she actually has/had MS. No MRI. If you look for it hidden in her webpage, you will find it – it the lack there of. Also, it’s not mentioned in any of her books. And don’t confuse her use of the word “heal” with “cure”. She states that her stuff isn’t a fix, buried deep in her website, I’m sure it’s for legal purposes that it has to be there, but it’s a bugger to find. Also buried deep but is there is that she still has symptoms and struggles, her magic diet is not a cure.
      It’s not unusual for people with relapsing-remitting MS to improve after a severe attack (me being one where I was paralyzed on my right side and left lower leg, I couldn’t walk and could barely even swallow along with the other hellish symptoms that are too long to list) even without meds, diets, or whatever, that’s the nature of relapsing/remitting.

      That being said, being as healthy as possible can only help and certainly won’t hurt.

  6. Debra says:

    While there is the endless “investigating” we are losing our lives. They say they don’t know what causes it, so how can a cure be found if the cause is not known?

  7. Christopher says:

    Helene,

    You could, but why? Hundreds of scientists are already working on stem cell therapies, but none are anywhere close to being ready to help anyone in any meaningful way. Besides which, I challenge anyone to provide actual proof that any company (or any group) is purposely holding any therapies back to keep people forever disabled, and thus exploit them for lifelong profit. It makes no sense, as MS is unpredictable and unable to be controlled–which includes making it a chronic disease instead of curable, without killing patients (not good for business).

  8. Karen Beaumont says:

    I have PPMS and hate my walking stick, and look older than my 59 years when I use it. There is a drug available (Orevus) but I can’t have it due to the cost. Why are drug companies so greedy when life has a cost?

    • Christopher says:

      Karen,

      Where are you located? There may be ways to help you get the medication anyway, even if you can’t afford it. But one of the most important factors is where you live.

  9. Reni says:

    “May” “in development” “promising”
    Lovely words they keep feeding us as we get worse and our quality of life is reduced to zero.

  10. Christopher says:

    Hi Collin. Definitely.

    I am in the US, and I see many people who post on here are from the UK. In the United States there are more than a few programs and organizations that help with access to medications for people with limited means. In the twenty-first century it’s tragic that this isn’t more accessible worldwide, when we all live in a highly interconnected world, and multiple sclerosis itself doesn’t recognize borders.

    I personally have been teaching myself to advocate and be of service to help more people with MS–such as myself–who aren’t sure or aware of where to go for knowledge, assistance, and resources related to life with MS. Since we are all related by this dreadful condition, it makes sense both pragmatically and compassionately to share as much information and knowledge as possible with each other. This is a sort of real life ‘distributed computing’ of knowledge and fellowship that, can make a difference by anyone being able to tap into informational and sympathetic/empathetic resources widely. And I personally believe in sharing my compassion, tolerance and knowledge, though I also do not demand the same of anyone else.

    Whatever I can do to help, I’m here to do so.

    • Chrisg says:

      Getting the drugs you need for your ms is just as bad in the USA. have you never heard of the donut hole. Once you are on Medicare, drug companies will not help you with any drug cost. I have also tried applying for state aid and got nothing. Have to stop taking tecfidera. USA is just bad now No help.

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