CRISPR: Making the Cut to Restore Vision

CRISPR (also known as CRISPR-Cas9) is a gene editing technique that allows scientists to cut DNA very precisely. It’s like a pair of molecular scissors that can be used to cut mutations out, or add new pieces of DNA into a gene. The importance of this tool was
highlighted in October 2020 when Dr. Emmanuelle Charpentier (Max Planck, Berlin) and Dr. Jennifer Doudna (University of California, Berkeley) were awarded the 2020 Nobel prize in Chemistry for this discovery.

CRISPR is actually a natural part of a bacteria’s immune system. It was discovered by Dr. Charpentier in 2011 and soon after, working with Dr. Doudna, showed that it could be used to cut any piece of DNA very precisely and at specific sites.

It has been less than 10 years since its discovery but CRISPR has already had a huge impact on biomedical science and is the basis of new treatments being developed and tested in clinical trials for many diseases including cancer and blinding eye diseases. For example, a CRISPR-based treatment is being tested in a clinical trial for Leber congenital amaurosis (LCA), an inherited retinal disease (IRD) that causes sever vision loss at birth. There are other potential treatments in development for IRDs and other eye diseases such as glaucoma as the study below explains.

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