Researchers at the Novartis Institutes for BioMedical Research (NIBR) report discovered a molecule that increases production of survival motor neuron (SMN) protein in cell and animal models. The discovery was published online in Nature Chemical Biology on June 1. Lack of SMN is seems to be the major cause of spinal muscular atrophy (SMA).
They’ve also demonstrated how the compound—which is being tested in a clinical trial in Europe—might work.
“Our compound can potentially boost SMN production by coaxing complex cellular machinery known as the spliceosome to help out,” says Susanne Swalley, who is a co-first author on the paper and an investigator in Developmental & Molecular Pathways at NIBR.
You can read more on their discovery at NIBR blog.
They’ve also demonstrated how the compound—which is being tested in a clinical trial in Europe—might work.
“Our compound can potentially boost SMN production by coaxing complex cellular machinery known as the spliceosome to help out,” says Susanne Swalley, who is a co-first author on the paper and an investigator in Developmental & Molecular Pathways at NIBR.
You can read more on their discovery at NIBR blog.
Comments
Post a Comment