Design and application of novel photoreceptor-specific promoters for retinal gene therapy applications
OwnerDavid Gamm, Yashdeep Phanseinventors
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Abstract
Provided herein are genetically engineered promoter-enhancer constructs for altering transcription of a gene operably linked thereto having specificity for expression in photoreceptor cells. Also provided are methods of use for these photoreceptor-specific promoter-enhancer genetic constructs; a pharmaceutical composition comprising these constructs, and methods of using the pharmaceutical composition for treating retinal diseases.
Claims24
A genetically engineered promoter-enhancer construct comprising a promoter and at least one enhancer operatively linked to the promoter that specifically promotes expression in a photoreceptor cell of a nucleic acid that is operatively linked to the promoter-enhancer construct and capable of being expressed therein.
Dependencies are inferred from the claim text.