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Home » Research News

Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy

by Kathy Jones on July 28, 2011 at 7:35 PM Research News

People with devastating genetic disease Duchenne muscular dystrophy were found to be benefited by a molecular technique originally developed at the University of North Carolina at Chapel Hill.

Duchenne muscular dystrophy is a lethal disease that affects 1 in 3500 newborn boys.

Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy

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In the disease, omissions or misprints in the letters of the dystrophin gene cause its "reading frame" to shift, abbreviating the instructions for making the dystrophin protein.

As a result, the cells fail to make a functional muscle protein and patients eventually lose their ability to walk and breathe.

In a study, researchers from the U.K., U.S. and Australia demonstrated that a phase Ib/IIa trial of the approach restored production of the critical muscle protein missing in patients with the progressive neuromuscular condition.

"When I first tried my approach in a test tube some twenty years ago, a reviewer of my manuscript commented that it was 'molecular gymnastics that will never amount to anything,'" said study co-author Ryszard Kole, PhD, a professor of pharmacology who develop the technology with AVI Biopharma, in Bellevue, Washington.

"Now we have evidence that it works, and in an illness that has no other good therapeutic options," he stated.

The novel treatment uses strips of genetic code - called antisense oligonucleotides - to restore the function of a defective dystrophin gene.

The scientists administered the treatment intraveneously (IV) to 19 Duchenne muscular dystrophy patients over the course of 12 weeks.

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If you have a question about health related issues, you can now post it in our Ask An Expert section on our community website Medwonders.com and get answers from our panel of experts.

saadi

12/05/2011

my son is involved DMD and his age is 11years ,now his movement is limited .he feel very very diffcult to walk even fell down.i m worry his future. please give me guidance of treatment.

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Mido-93

09/21/2011

hi I'm Dr Menat , I'm from Egypt and my son is suffreing from DMD . has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD? . and Is this treatment for all types of mutations that occur in DMD or for one type only ? . I'm looking forward to hearing your reply soon . Don't forget me .

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Sushma_Gupta(Guest)

09/08/2011

is there any chances of improvement in conditions of patients suffering from DMD??????????

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25774230

09/06/2011

hi i am pradnya gaikwad has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD?

Reply | Forward

karapinar(Guest)

08/27/2011

has this actually been carried out and found to be successful and is is there any possibility of it becoming a near future treatment for DMD?

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Treatment of Duchenne Muscular Dystrophy may be Possible Via Molecular Therapy

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