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Thread: Theory on How to Cure and Repair Muscle Loss Caused by Spinal Muscular Atrophy

  1. #1 Theory on How to Cure and Repair Muscle Loss Caused by Spinal Muscular Atrophy 
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    Apr 2019
    Hello, just for background information I'm a 15 year old female who's a sophomore in high school, and the most complex science class I've taken thus far is Honors Bio. Therefore, I'm fully aware that my theory on how to cure this genetic condition is drastically oversimplified and I hope to be able to add on and revise my theory as I take more complex sciences (AP Biology next year!!).

    My little brother in fact has Spinal Muscular Atrophy - he's a type 3 - and I as well as both of my parents are carriers for this condition. For a quick run-down on how this condition works if you've never heard of it, essentially my brother either inherited two broken copies of SMN1 or he didn't inherit any copy of SMN1 at all. SMN1 produces the SMN protein, and if you don't produce this protein in any capacity you are incompatible with life. You only need one copy of SMN1 to be alive, which is why my parents and I are able to be fully functional despite lacking a second copy of SMN1. However, there's a backup gene known as SMN2, which doesn't NEARLY as much SMN protein as the SMN1 gene, but it does allow those lacking the SMN1 gene to be compatible with life. Depending on how many copies you have, your SMA is less severe. My brother has 4 copies of SMN2 (you can have many copies, not just two at max like you do with SMN1), thus he's a type 3 a.k.a. the least severe of the three types.

    He's projected to live a normal lifespan, however since he doesn't produce much of the SMN protein his motor neurons are unable to communicate with his muscles efficiently and tell them to move. Since muscles need to be stimulated otherwise they die off, my brother's muscles have progressively atrophied as the years have gone by. He's turning 13 in 10 days, so he's had 13 years of muscle damage! Despite him having the least severe form of SMA, we estimate he's lacking anywhere from 30-40% of the muscle mass a boy his weight, height, and age typically has. So while it may be noted the least severe of all the types, it still causes serious muscle loss in the long term. There's currently a drug known as Nusinersen (or it's more common commercial name you may have heard of - Spinraza), which works to stabilize the levels of SMN protein in the body. However, this only prevents further muscle degradation, and doesn't repair the 13 years of muscle atrophy my brother has experienced, as well as his broken motor neurons. I'm proposing what I believe, in theory, would not only cure SMA, but would also provide a course of treatment to repair the muscle loss experienced by those with SMA that have experienced muscle degeneration over the years.

    The first phase of this would involve the idea of producing a sort of synthetic muscle cell. This would act as to not only regenerate the lost muscle tissue, but also to eliminate the old muscle cells. I'd rather use a synthetic muscle cell to reproduce the lost muscle tissue instead of the old ones as the old muscle cells have already experienced telomere degradation due to having undergone mitosis countless times in a time span of, say, 13 years. I conceptualize that if one were to inject the DNA of said patient into an empty muscle cell, alter a TINY sequence of DNA that is rubbish DNA in the cell such as converting a AGCG chain into a TGCG chain, make a clump of said cells to form a sort of blastula-like cell to allow for the cells to rapidly divide until they hit equilibrium and stop undergoing such frequent mitosis, not only would the muscle damage be restored but the possibility of cancer would hopefully be eradicated as blastula cells of course do stop dividing so rapidly once said cells hit a certain frequency in the body. I see this being better than STEM cells, at least in this particular instance, because there is an eminent threat of cancer from STEM cells, and of course for someone with SMA that would only worsen their condition.

    You may be wondering, but what about the old muscle cells? This is why a tiny sequence of DNA must be altered in the replacement synthetic cells - as the blastula divides, the only way to once again prevent cancer and too much interaction between old and new muscle cells would be to use a virus that specifically targets said AGCG chain that would only create a lock and key fit to muscle cells. Since they'd be detecting for this specific chain and can't bind to any other human body cells, said virus would eradicate the old muscle cells as new ones emerge at the same pace to prevent both further degradation of the muscle tissue and to prevent, once again, cancer from occurring during treatment.

    Great! Now we'd have new muscle cells and repair the damage. But how do we make SURE this damage doesn't occur again? This would be achieved via gene therapy to give a working copy of SMN1 to the patient, which would make sure they have the correct amount of SMN protein in them at all times, as well as repairing the motor neurons to make sure they can adequately communicate with the muscles as they would normally communicate with each-other in one that doesn't have SMA. In theory, synthetic cell treatment and gene therapy would cure and repair a patient with SMA's muscle loss they'd experienced with their days living with the disease. And, if the SMN protein needs to be regulated, we already have Nusinersan which does such thing.

    I understand that this is EXTREMELY hypothetical and would be incredibly expensive and time-consuming to produce, but I really would like opinions if this is at least feasible and if I should continue researching my theoretical cure further. SMA is such a nasty disease - it's taken so many lives away from this Earth far too early, and I'd love to see said disease be eradicated in my lifetime. Please give me your feedback, it'd be greatly appreciated!

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