One more step in gene therapy

A new therapeutic pathway for diseases based on genes is opened. Scientists at Imperial College London and Hammersmith Hospital collaborate in the process of protein formation to correct errors.

The research focuses on the cells of a patient with muscle atrophy. A specific gene for these cells is defective, so it does not form the proper protein, since this failure affects the short one.

To solve this they have used small parts of modified RNA, the oligos. They are able to adhere to exons involved in protein formation and recover the proper expression of faulty genes.

It is not the first time that oligos are used, but so far they have only been used to block the expression of genes and not to adequate expression.

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