Molecular Cell·· 7 hours agoPickScore76
Regulatable in vivo gene expression via adaptamers.
In brief
Researchers developed the adaptamer, a compact RNA switch pairing FDA-approved small molecules with endogenous ADAR-mediated RNA editing to reversibly control transgene expression in vivo. The element is under 120 bp and needs no exogenous protein machinery; AAV-delivered FGF21 expression induced metabolic remodeling in mice, reversing obesity and hepatic steatosis.
Why it matters
The paper gives the under-120 bp size and mouse functional data with AAV-delivered FGF21, letting readers compare with existing inducible systems on size, low immunogenicity, and use of FDA-approved small molecules.
Source: Molecular Cell