At Avellino, we are in the process of developing advanced gene therapies for corneal dystrophy using CRISPR-Cas9.
What is CRISPR
CRISPR is a gene editing tool that is modeled off a naturally occurring phenomenon in bacteria. The acronym stands for “Clustered Regularly Interspaced Short Palindromic Repeats,” which are repeated segments of DNA. Bacteria will locate one of these repeated segments of DNA in an invading virus and cut it out using an enzyme called Cas9. This disables the virus and stores the genetic information to identify the virus again in the future. Now, scientists are developing synthetic CRISPR-Cas9 to identify and remove sections of DNA that cause inherited diseases in humans.
Gene Editing at Avellino
Avellino’s Chief Research and Innovation Officer Dr. Tara Moore is a professor at Ulster University in Northern Ireland, where their Research Partnership Program has been working on finding a treatment for corneal diseases via CRISPR gene editing, guide RNA design, and in vitro and in vivo gene editing and treatment in animals and humans.
To direct the focus of this research onto corneal diseases, Moore’s team began using Small interfering RNA (siRNA), or “silencing RNA.” These siRNA are non-coding and interfere with, or silence, the expression of specific genes like those responsible for developing corneal disease. One of the Research Partnership Program’s latest achievements is joint research with SiSaf Ltd. focused on the development of siRNA drug delivery technology using nano particles, and a second project focused on editing of the TGFBI gene via CRISPR-Cas 9.
This research may lead the way toward treating select corneal dystrophies, potentially improving eyesight for millions.