(IMAGE)
Caption
We designed and synthesized both a conventional ASO and a ASO modified with BNAP-AEO (BNAP-AEO ASO) (top row). The ASO with BNAP-AEO exhibited a higher binding affinity to the target RNA than the conventional ASO (middle left). Additionally, the BNAP-AEO administered into mice intrathecally showed lower neurotoxicity compared to the conventional ASO without BNAP-AEO with evaluation by toxicity score as well as assessment of spontaneous motor function (open field test), while maintaining the inhibitory effect on the target gene (middle right). In other words, BNAP-AEO demonstrates high binding affinity, efficacy, and safety simultaneously.
Credit
Department of Neurology and Neurological Science, TMDU
Usage Restrictions
None
License
Licensed content