SpliceR

SpliceR is a gene editing tool that utilizes an exon skipping approach to modulate RNA splicing. This technology is designed to help cellular machinery skip over specific exons in genes, which can be beneficial for treating diseases caused by mutations that result in misfolded or toxic proteins. In preclinical studies, SpliceR has demonstrated the ability to efficiently reduce the formation of amyloid-beta plaque precursors in mouse models of Alzheimer's disease by targeting and skipping specific exons within relevant genes. The platform shows promise for broader application in other conditions such as Duchenne muscular dystrophy and Huntington's disease, where exon skipping could mitigate pathogenic protein production[4].

Brand names

SpliceR

Other names

SpliceR