Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

Helmholtz Munich and the University of Sydney have identified biological mechanisms that are shared across psychiatric ...

That loss can be compensated for by a related gene, called SMN2, which produces smaller amounts of the protein. The more copies of SMN2 a person has, the milder the symptoms are likely to be.

The antisense oligonucleotide works by modulating alternative splicing of the SMN2 exon which is mutated in the disease. This is however "laborious, slow and costly", according to the biopharma.

The cranial nerves are a set of twelve pairs of nerves that travel to and from the brain. Each has a different function. For example, the olfactory nerve is essential for detecting smells. The ...

who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. Breaking barriers to CAR T-cell therapy: The vision for expa... Live from ASCO 2025 ...

For our high-throughput screening campaign, we utilized an NSC34 motor neuron cell line expressing an SMN2 minigene reporter ... via qPCR for dose responsiveness and to confirm the desired splicing ...

Alternative pre-mRNA splicing is a fundamental mechanism of post-transcriptional gene regulation that creates lineage-specific functional diversity in the proteome. Although heart-specific alternative ...

Abstract: A key challenge of image splicing detection is how to localize integral tampered regions without false alarm. Although current forgery detection approaches have achieved promising ...