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Empowering Retinal Gene Therapy with a Specific Promoter for Human Rod and Cone ON-Bipolar Cells

Elmar Carlos Hulliger, Simon Manuel Hostettler, Sonja Kleinlogel

2020Molecular Therapy — Methods & Clinical Development52 citationsDOIOpen Access PDF

Abstract

mouse model of late retinal degeneration. The new promoter enables precise manipulation of the inner retinal network and paves the way for clinical application of gene therapies for high-resolution optogenetic vision restoration, raising hopes of significantly improving the life quality of people suffering from blindness.

Topics & Concepts

RetinalGenetic enhancementCone (formal languages)GeneRetinaBiologyMedicineGeneticsOphthalmologyNeuroscienceComputer scienceAlgorithmRetinal Development and DisordersPhotoreceptor and optogenetics researchNeuroscience and Neural Engineering
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