06 November 2015

CRISPR/Cas9 system will be used to treat Leber's amaurosis

Spot editing of the human genome was promised to begin in 2017

Anna Obraztsova, N+1 

Bill Gates-backed biotech startup Editas Medicine will begin clinical trials of CRISPR/Cas9 technology for targeted editing of the human genome over the next two years. This was stated by the CEO of the company, Catherine Bosley at the EmTech conference, her words are transmitted by MIT Technology Review (CRISPR Gene Editing to Be Tested on People by 2017, Says Editas).

Editas plans to correct errors in genes that cause hereditary human diseases. For the tests, the specialists chose one of the forms of a rare eye disease – Leber's amaurosis, in which the light-sensitive cells of the retina die off up to complete loss of vision. This disorder occurs in only one newborn out of 80 thousand, but it is successfully suitable for treatment with gene therapy. Firstly, a defect in a certain gene that causes the disease is already known, and secondly, the eye is a fairly convenient organ for drug delivery. The therapy will be an injection into the retina of a solution of viruses carrying DNA necessary for the production of the CRISPR/Cas9 system in diseased cells. About a thousand nucleotides will be removed from the CEP290 gene, after which it should start functioning normally.

The basis of the CRISPR/Cas9 system is the Cas9 nuclease, which replaces the damaged DNA fragment with a normal one, and the guiding RNA molecule, which allows the enzyme to accurately determine the place in the genome where changes need to be made. With the help of such a system, the editing of "broken" genes becomes relatively cheap and simple, which makes it a promising tool for medicine. However, the use of CRISPR/Cas9 on humans has not yet been tested (with the exception of the scandalous experiment of Chinese scientists on non-viable embryos).

The development and clinical trials of such therapy require significant funds. In August, Editas received $ 120 million from private investors, and the main one of them is Bng0, one of the participants of which is Bill Gates.

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