Abstract
Provided herein, inter alia, are expression systems capable of modulating expression of a target gene. In embodiments, the expression systems silence the expression of an endogenous version of the gene and induce expression of a recombinant version of the gene. The expression system provided herein is contemplated to be effective for treating genetic disorders.
Full Text
What is claimed is:
Provided herein, inter alia, are expression systems capable of modulating expression of a target gene. In embodiments, the expression systems silence the expression of an endogenous version of the gene and induce expression of a recombinant version of the gene. The expression system provided herein is contemplated to be effective for treating genetic disorders.
Timeline
Filed
04/01/2026Published
07/23/2026Granted
Not AvailableIPC Codes(6)
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 31/7105:Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 38/17:from animals; from humans