Duchenne muscular dystrophy

Gene Therapy

This attempts to alter defective alleles in the affected tissues.
Method is therapeutic for the patient only.
Genetic changes are not inherited.

This attempts to alter the defective allele in germ line cells (oocyte).
These corrections in the genome could be inherited.
Rare and contoversial due to interactions between genes.

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Symptoms

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Gene therapy

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Dystrophin protein

DMD is caused in 1/3500 births by a recessive, sex linked mutation of the dystrophin gene.

The body fails to make dystrophin, a structural component of muscle tissue.

This leads to progressive muscle wastage resulting in wheelchair use by 12 and a shortened life span.

Gene therapy of cystic fibrosis involves the CFTR gene being introduced via liposome vectors into the affected tissue (lungs) and the lung cells then begin to make the absent protein alleviating the symptoms.

In DMD

The gene is too large to be carried by virus or liposome vector.
There is a large amount of affected tissue in the body (all muscle tissue).
DMD sufferers run the increased risk of dangerous immune responses.

The dystrophin gene is coded for by 79 exons.

This makes a large protein.

The middle of this protein contains a large number of repeated segments.

In a disease called Becker muscular dystrophy some of the protein segments are missing and relatively mild symptoms are seen.

BIOLOGY