CRISPR Technology in Human Gene Editing: Are We Ready For It?

With the new gene editing technique called CRISPR, damaged DNA could be repaired. “CRISPR technology is incredibly powerful and could be extremely helpful and useful,” said Nancy Wexler, professor of neuropsychology at Columbia University and member of a National Academy of Sciences panel coming up with possible new rules on CRISPR. However extremely useful, CRISPR can cause too many off target effects. It wouldn’t be wise to use it if by using it we are curing one disease but causing another. So that raises the question if we are ready for prime time.

Editas CEO Katrine Bosley, from Mass.-based startup firm Editas Medicine, Cambridge said at a conference that a CRISPR -- which stands for clustered regularly interspaced short palindromic repeats, essentially segments of DNA containing repeated base sequences -- can prove to become a promising cure for treating inherited disorders that are the result of “broken genes,” for instance- Huntington’s disease or cystic fibrosis. The company says that it wants to run a clinical trial for Leber congenital amaurosis (LCA) which targets the retina and affects about 600 people in the United States.

The cure would involve injecting the genetically engineered viruses into the photoreceptor cells of the retina. The protein Cas9 would then cut away the damaged part of the DNA and the healthy DNA would rejoin.

CRISPR’s off target effects include snipping the genes that aren’t targets. Although it works very perfectly, sometimes it may happen to make a cut on a gene that is working to protect body from a particular disease and that is something we certainly do not want. So while using CRISPR, we have to make sure it is not interfering in body’s natural pathways.