RNATACs: Multispecific small molecules targeting RNA by induced proximity.
Cell Chem Biol
; 31(6): 1101-1117, 2024 Jun 20.
Article
en En
| MEDLINE
| ID: mdl-38876100
ABSTRACT
RNA-targeting small molecules (rSMs) have become an attractive modality to tackle traditionally undruggable proteins and expand the druggable space. Among many innovative concepts, RNA-targeting chimeras (RNATACs) represent a new class of multispecific, induced proximity small molecules that act by chemically bringing RNA targets into proximity with an endogenous RNA effector, such as a ribonuclease (RNase). Depending on the RNA effector, RNATACs can alter the stability, localization, translation, or splicing of the target RNA. Although still in its infancy, this new modality has the potential for broad applications in the future to treat diseases with high unmet need. In this review, we discuss potential advantages of RNATACs, recent progress in the field, and challenges to this cutting-edge technology.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
ARN
/
Bibliotecas de Moléculas Pequeñas
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Cell Chem Biol
Año:
2024
Tipo del documento:
Article
Pais de publicación:
Estados Unidos