Cancer is caused by faulty genes, but what also shapes a cancer cell's behavior is how a gene's instructions are trimmed and ...

Researchers have identified a potential treatment strategy for Rett syndrome via alternative splicing modulation of the MECP2 gene.

Proteoforms, the diverse molecular variants of proteins, are key to understanding cellular functions, disease mechanisms, and ...

More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

Drug-resistant epilepsy affects millions worldwide, with focal cortical dysplasia (FCD) as one of the leading causes. Yet its ...

When Associate Professor Eliezer Calo PhD '11 was applying for faculty positions, he was drawn to MIT not only because it's ...

An important study has uncovered an unexpected molecular player that helps make cancer cells stress-resilient, thereby promoting the survival, growth and progression of tumours. Researchers have ...

Houston Methodist scientists have uncovered an unexpected side of a protein best known for its link to brain diseases. The ...

ARTHEx Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast ...

CRF awards four global grants to advance CHM research toward new clinical trial endpoints and therapeutic approaches—driving progress toward treatments.

Over the past decade, however, a wave of technological advances has begun to expand the druggable proteome. These include covalent inhibitors (such as sotorasib), chemical proteomics, and AI-enabled ...

PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...