Behnam Rabiee

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Eye-opening treatment for haploinsufficiency Aniridia is a genetic disorder predominantly affecting the eye, caused by PAX6 haploinsufficiency due to mutations in one allele. Patients present vision loss and, in some cases, other systemic abnormalities. Increasing the expression of PAX6 early in life could block the progression of the disease. Now, Rabiee et al. used a pharmacological approach in vitro and in a rodent model of Pax6 haploinsufficiency (Pax6+/−).