One idea has been that speckles are splicing factories. The splicing reaction would happen inside the speckle, and then the spliced product would leave. “What people found is that’s not what happens,” ...

Human genes that encode proteins often contain non-coding segments known as introns. Removing introns is crucial for the proper expression of genetic information. Understanding how our cells ...

Preclinical in vivo data demonstrate hyper-precise mechanism directly targets the disease-causing mRNA repeats leading to correction of splicing ...