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Gene therapy to self repair method of cells

Gene therapy experts in Germany have come to the conclusion that cells can repair themselves without replacing a faulty gene. The new approach controls the cells’ own correctional system with a drug influence by stimulating a related defective gene.

The research was focused on spinal muscular atrophy (SMA), in which the survival motor neuron gene (SMN1) is erased. Its copy gene produces only ten percent of correct protein. The researchers experimented with a drug called valproate. It was found later that it could increase levels of the SMN protein up to four times. It also raised SMN levels in brain tissues.

The research team first treated 10 parents of children with SMA for four months. The drug valproate increased SMN levels in blood and then 20 SMA patients were treated of which seven responded positive. It showed clearly that influencing the behavior of genes with drugs is possible. The researchers hoped that this research may produce noteworthy benefits in the future.

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