While humans can escape the heat by seeking shade or shedding layers, plants remain rooted in place. So how do they survive ...

Spain’s SpliceBio will use $135 million from a series B round backed by Sanofi and Roche to advance its lead gene editing therapy for Stargardt disease through clinical studies. | Spain’s ...

Provided herein are novel heterocyclic compounds as SMN2 modulators, pharmaceutical compositions, use of such compounds in treating spinal muscular atrophy (SMA) via modulation of SMN2 splicing, and ...

Researchers have taken preliminary steps toward treating a devastating neurodevelopmental disorder, spinal muscular atrophy, with a genetic therapy delivered in the womb, before the worst damage ...

Gene conversion from SMN1 to SMN2, which causes spinal muscular atrophy (SMA), appears to be a key genetic characteristic of the disease, a study found.

Risdiplam is an SMN2 pre-mRNA splicing modifier designed to increase and sustain SMN protein levels centrally and peripherally.

SMN2 copy number can carry great implications for patients with SMA. The majority of SMA cases result in the absence or loss of SMN1, leaving patients and their motor neurons to relying on SMN2.

Johns Hopkins researchers have developed a powerful new AI tool called Splam that can identify where splicing occurs in genes—an advance that could help scientists analyze genetic data with greater ...

Both molecules modify a process called splicing in the SMN2 gene. Combining multiple splice-modifying molecules, including Evrysdi with branaplam, appeared to have synergistic effects that could make ...

Ravindra Singh, professor of biomedical sciences, led a research team that created a shortened version of the gene that cause spinal muscular atrophy. The super minigene will make it easier to find ...