Nine years ago, scientists discovered that bacteria use a collection of genetic sequences and an associated enzyme as part of their defense against invading organisms, such as viruses. This system, called CRISPR-Cas9 (pronounced crisper-cass nine), remembers, recognizes, targets, and then cuts viral DNA, rendering it ineffective. Over the last nine years, CRISPR-Cas9 has been engineered to precisely target and cut DNA as a means of treating diseases caused by genetic mutations.

PhD student Dr. Andrea Minella and Dr. Simon Petersen-Jones of the Comparative Ophthalmology Lab are now exploring the use of CRISPR-Cas9 for treating cats with a particular form of Progressive Retinal Atrophy, an inherited disease that causes blindness. The concept is simple: direct the CRISPR-Cas9 system to cut out the mutated region of the implicated gene and replace it with a “normal” bit of DNA, thereby correcting the disease-causing mutation.

The execution, however, is far more complex and still being developed at the cellular level. Drs. Minella and Petersen-Jones are optimistic that this can be done, and could be used to treat humans with Leber Congenital Amaurosis.