CRISPR in the clinic


CRISPR-Cas9 is a genetic tool that can perform molecular surgery on the DNA of a cell. Fundamentally altering the code of life allows scientists to insert and remove genes.

For the first time in a milestone study, CRISPR-Cas9 has been implemented in a clinical setting to alleviate blindness in an untreatable genetic condition known as Leber’s congenital amaurosis.

A single participant with Leber’s congenital amaurosis 10 (LCA10), has been administration CRISPR-Cas9 through injection into the eye near photo-receptor cells (which detect light and color). If successful it may reverse the disease by removing a single mutation in the DNA - allowing sight.

This form of treatment is considered safe since CRISPR-Cas9 stays locally in the eye, and therefore should not travel to other sites of the body.

The estimated time-frame is a month before vision is restored. Success of this trial may result in 18 more patients commencing with treatment in the next trial phase.

Mark Pennesi from Oregon Health & Science University in Portland said to Nature - 'This is one of the few diseases where we think you could actually get an improvement in vision'.

This is a historical moment for genetic editing and life on earth. If insertion of CRISPR-Cas9 into the body proves successful, the gene editing tool will be considered to harness unparalleled potential. Paving the way to address the estimated 1 in 2000 world-wide suffering from single gene retinal inherited mutations.






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