CAS: 1449598-06-4

RG7800

RG7800 is a small molecule inhibitor, which is a product synthesized through advanced organic chemistry processes. Its mode of action involves the selective binding to specific proteins within cellular pathways, effectively inhibiting their function. This is achieved through high-affinity interactions that alter the conformational states of targeted molecules, thereby impeding their biological activity. RG7800 is primarily utilized in the study of genetic regulation and therapeutic interventions. It is extensively applied in research focusing on the modulation of RNA splicing and has shown potential in therapeutic applications for diseases linked to aberrations in RNA processing. This compound is particularly valuable for exploring the molecular mechanisms underlying neuromuscular disorders, providing insights that drive the development of targeted therapies. By facilitating the study of specific molecular interactions, RG7800 aids in elucidating the intricate dynamics of cellular processes, advancing both basic science and applied biomedical research.

RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier.