Characterization of Ribozymes Targeting a Congenital Night Blindness Mutation in Rhodopsin Mutation.

Abstract excerpt

The G90D mutation in the rhodopsin gene leads to autosomal dominant congenital stationary night blindness (CSNB) in patients. This occurs because the G90D mutant protein cannot efficiently bind chromophore and is constitutively active. To combat this mutation, we designed and characterized two different hammerhead ribozymes to cleave G90D transcript. In vitro testing showed that the G90D1 ribozyme efficiently and...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Biocatalysis
• Share to Dependovirus
• Share to Eye Diseases, Hereditary