Congenital aniridia is an inherited eye disease that affects all parts of the eye, including the cornea. It leads to progressive corneal clouding and, eventually, blindness.
Current treatment involves the transplantation of corneal stem cells from a deceased donor, which requires aggressive immune suppression.
Our project aims to develop personalised treatment strategies that eliminate the need for immune suppression and correct the underlying disease mechanism by addressing the genetic mutation.
Patient enrolment has begun and is ongoing. We have successfully transformed patient skin cells into stem cells, which will then be converted into corneal stem cells using existing protocols.