New Breakthrough in Cure For Muscular Dystrophy

March 17, 2009

(ChattahBox)– Scientists may be getting closer to finding a cure for Duchenne Muscular Dystrophy and other devastating genetic disorders with the groundbreaking application of exon-skipping DNA molecules, which skip over the mutated gene acting as a genetic band-aid. The encouraging results of an ongoing study were just published in the Journal of the American Neurological Association. Genetic researchers from the Washington, DC based Children’s National Medical Center and the National Center of Neurology and Psychiatry in Tokyo, jointly conducted its first successful trial of the DNA molecule therapy in dogs, with promising results for millions of MD sufferers and their families around the world.

Duchenne Muscular Dystrophy is a devastating X-linked genetic disorder, which blocks the creation of dystrophin needed to form muscles. Other lethal, genetic muscle disorders fall under the general category of Muscular Dystrophy, such as the heartbreaking Spinal Muscular Atrophy, or SMA, which is the Number One genetic killer of infants under the age of two.

This exciting clinical study is important for two reasons. It first proves that the application of exon-skipping gene therapy is possible in a large-organism, such as a dog. Previous studies were only successful on mice. Scientists call this type of treatment systemic anti-sense therapy. In clinical terms, this study proves “proof-of-concept” that this new gene therapy will one day work on humans afflicted with MD. Secondly, and more importantly, the gene therapy worked! The dogs treated with the exon-skipping therapy showed clinical improvement in their MD symptoms.

The afflicted dogs were treated with a multi-range cocktail of the antisense therapy to reach all 79 possible mutated genes in a patient with MD. The result was extremely promising. Scientists found that the cocktail skipped over multiple mutated genes, making this therapy effective for the majority of MD patients.

Furthers studies of course, need to be conducted before clinical trials can be developed on humans. In addition, it’s extremely expensive, at the moment, to administer, as the therapy requires large doses of the antisense molecule into the blood. In addition, the dog’s hearts, also affected by lack of muscle protein, did not improve with this therapy.

These early studies are an exciting development in the application of exon-skipping gene therapy to treat Duchenne Muscular Dystrophy and other genetic disorders.


8 Responses to “New Breakthrough in Cure For Muscular Dystrophy”

  1. Colonel Amarjit Singh Retired on March 18th, 2009 1:02 am

    I am very excited to read such news. Well done SIR. Perhaps I can, very anxiously, look forward to see my 8 years old grand son get treated for DMD. He is suffering from duplication of EXONE 2. I wish to know as to HOW soon it would be possible to treat my grand son.

  2. Sue on March 18th, 2009 5:04 pm

    Hi Colonel Colonel Amarjit Singh:

    Thank you for your comment and your interest. I am so sorry to hear about your grandson with DMD. I know firsthand how devastating it is to watch a loved one suffer with a genetic muscle disease. My own grandson recently passed away from SMA type 1, so as you can imagine this issue is dear to my heart.

    I wish I had an answer for you regarding when such a treatment would be available. I suggest you frequently check the MD website to keep abreast of new clinical trials. Also, stay tuned to ChattahBox for frequent health news.

    I pray your grandson will one day be able to receive this treatment.

    God Bless.



  3. ruchika batra on March 18th, 2009 11:23 pm

    I am a parent of a 6 year old with DMD. He has deletion of exons 49 and 50. Since 51 skipping will make the deletion ‘in-frame’ and advanced studies for skipping exon 51 are under way. Would you please give me a time frame as to when the therapy could be available for my son. This is ofcourse if every thing goes well at each stage…..

  4. Ritesh Sehgal on March 19th, 2009 3:06 am


    Many congratulations for this great development in Gene Therphy. Such news really encourges us to fight on as my only 1 year old daughter has Spinal Muscular Atrophy Type 1. I am saving every money for donations of such clinical trails and for my daughter’s future treatment.
    Sir could please let me know as about what time can we expect a breakthorugh under this and whether it would help SMA type 1.

    Many Regards and thanks

    Ritesh Sehgal
    A-160 , Defence Colony
    New Delhi – 110024
    +91 9818099200

  5. M.Hakim Farid on March 30th, 2009 1:13 am

    I am fighting every day with the creater of the world as I see my two daughters conditions going from bad to worse however they are 14 and 13 years old and it seems that God is hearing their mother prayers,could you tell me that how soon it will be possible for my daughters to go back to school .

  6. Wendy McKinley on April 24th, 2009 12:40 pm

    This is very encouraging, I am thankful for each day that my son can still walk.He has Becker Muscular Dystrophy. Although I see him struggle to lift objects that weigh hardly anything and I watch his awkward gait, I remind myself, he is still walking. My hope is that the cure will get here in time, he is already 26 years old. I feel as though I’m holding my breath each day, wondering if this will be the day he will no longer walk.
    Thank you for all your hard work, I know that it is time consuming and very expensive.
    The sooner we find the cure, the more lives can be saved. Not everyone is as fortunate as my son, they do not have that much time on their side.
    Applauding your efforts,
    Wendy McKinley

  7. Mrs.H.H.M on July 23rd, 2009 9:02 am

    peace be onto you all

    Great to know that there are some good people doing their best to serve the pained humanity. May God guide them to the best cure.Amen and May God reward them for their efforts.Amen Also may these drugs or treatment be affordable and reach in time to to each and every individual in need.Amen

    I have two nieces suffering from MD from the past 20 years. Both of them are married and are of the age 29 and 27 respectively. Both of them have kids. The younger who is 27 has three kids, her eldest being 11, middle one 6 and youngest is 3.
    While the elder sister who is 29 has one son of the age of three.
    When I see these mother’s how they toil all day long on the wheel chair and their babies move around them crying for their lap, and the helpless mothers with the help of other elders and their own will power try to somehow sooth them feed them, hug them my heart aches. The children cant find comfort in any body’s arms but the lap of their moms.They are very much attached to them. They often sit down by their wheel chairs and pray with tears in their eyes. Sometime they would wonder and ask innocently why our mother cannot walk like other moms?
    It is so painful. They are not aware of the dangers of the their sickness.
    We prayed over the years and have gone to every place where they could be treated. But all in vain.

    My sister in grieve is bed ridden by seeing the condition of her daughters. But she is not not lost the hope.. She is every breath all prayerful for their health and waiting for a miracle. So do we.
    We wish now there should be a cure. An instant cure. Amen

    regards best wishes and prayers for all

    A Worried Aunt

  8. sreejakunnath on December 3rd, 2009 8:39 am

    im sreeja im also praying and eagerly waiting for a cure for MD. My sisters son is affected by DMD.. all of us are the sailors of the same boat let us pray together to find an immediate remedy… if any positive information, pls email to me sreeja.kunnath@gmail .com

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