Entrada Therapeutics has secured the Health Authorities and Ethics Committees' authorisation within the European Union (EU) ...

In four groups of boys with DMD (three boys in each group), an escalating ... of antisense oligonucleotides in the modification of SMN2 splicing in patients with SMA are likely to emerge.

In parallel, advances in oligonucleotide technologies for engineering SMN2 pre-mRNA splicing are approaching ... Previous studies with AONs in mouse models of DMD have shown relatively good ...

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

Early treatment in SMA provides benefits, but children with two SMN2 copies may still experience significant motor delays and disabilities. Infants with two SMN2 copies showed delayed motor ...

It is due to deletion (in 95% of cases) or gene conversion (SMN1 to SMN2) by nucleotide substitution at one point. Motor neurons of the anterior horn of the spinal cord. Credit: Jose Luis Calvo ...

Singh, a professor of biomedical science at Iowa State University, led an eight-year project to create a truncated version of the Survival Motor Neuron 2 (SMN2) gene to facilitate quicker ...

Determining the copy number of SMN1 and SMN2 genes is notoriously difficult due to the high level of homology between the two genes. These genes, that are associated with spinal muscular atrophy (SMA) ...

eGene Inc. has announced that a research article regarding their HDA-GT12™ was published in Clinical Chemistry's March 2006 issue titled "Determination of SMN1/SMN2 Gene Dosage by a Quantitative ...