ornithine aminotransferase-transduced keratinocytes

Ornithine aminotransferase-transduced keratinocytes represent an experimental ex vivo gene therapy approach designed to treat gyrate atrophy of the choroid and retina (GA), an autosomal recessive disorder caused by deficiency of the mitochondrial enzyme ornithine aminotransferase (OAT). The therapy involves harvesting autologous keratinocytes from the patient, transducing them with a viral vector (typically a retrovirus) carrying the functional human OAT cDNA, and then grafting these engineered cells back onto the patient. The goal is to create a metabolic "sink" where the grafted skin expresses high levels of OAT, thereby facilitating the systemic clearance of excess ornithine from the plasma and preventing the progressive chorioretinal degeneration associated with hyperornithinemia. This approach was pioneered by researchers at institutions like Johns Hopkins University as a form of somatic gene therapy to provide enzyme replacement without requiring systemic vector delivery.

Other names

OAT-transduced keratinocytesornithine aminotransferase-modified keratinocyteskeratinocytes transduced with human OAT cDNA