CRISPR Reactivation of the Fragile X Gene

CRISPR Reactivation of the Fragile X Gene

“We are trying to target the first event that goes wrong in fragile X syndrome”, says Todd, “One reason our previous attempts to develop treatments for fragile X syndrome have failed is that they’ve tried to target the downstream effects of losing the fragile X protein. The protein does many things… bypassing all the functions that it normally takes care of has proven difficult from a pharmacologic perspective.”

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CRISPR – Does it hold promise for Treatment of Fragile X Syndrome?

CRISPR – Does it hold promise for Treatment of Fragile X Syndrome?
There’s been a lot of press concerning a new biotechnology called CRISPR/Cas9, or simply CRISPR. This technology, which is based on the discovery of naturally-occurring bacterial defense mechanisms, has attracted an enormous amount of biotech investment. It has also excited the imaginations of scientists, clinicians, and rare disease advocates everywhere. How might CRISPR be applied to Fragile X syndrome? CRISPR offers the tantalizing possibility of “editing” genes very precisely, and it could (theoretically) excise the methylated trinucleotide repeat sequence from Fragile X cells, rendering them entirely normal. CRISPR: A Developing Technology First, it is important to understand that this is a developing technology. CRISPR is a powerful new research tool for editing genes; it can be used to enhance gene therapy approaches in vitro (in a dish in the lab), and to knock out genes quickly and easily. To date, its biggest advantage is the speed and cost-effectiveness with whichRead more