Abstract
Provided herein, inter alia, are expression systems capable of modulating expression of MECP2. In embodiments, the expression systems silence the expression of endogenous MECP2 and induce expression of recombinant MECP2. The expression system provided herein may be useful for treating a genetic disorder such as Rett Syndrome.
Full Text
What is claimed is:
Provided herein, inter alia, are expression systems capable of modulating expression of MECP2. In embodiments, the expression systems silence the expression of endogenous MECP2 and induce expression of recombinant MECP2. The expression system provided herein may be useful for treating a genetic disorder such as Rett Syndrome.
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 31/711:Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 38/17:from animals; from humans