by Researchers at Tokyo Medical and Dental University (TMDU) and Osaka University have discovered that a modified sugar significantly improves the safety and efficacy of Antisense Oligonucleotides (ASOs) in treating central nervous system (CNS) diseases.
CNS diseases, which include conditions affecting the brain and spinal cord, can be debilitating and require innovative treatment approaches. In a recent study published in Molecular Therapy Nucleic Acids, a team of researchers from Japan reported that a minor modification to an existing CNS disease treatment led to substantial improvements.
ASOs, a novel therapeutic strategy for treating CNS diseases caused by toxic proteins, have shown promise in clinical applications. These small pieces of genetic material bind to RNA messenger molecules that produce mutant disease-causing proteins, marking them for degradation. Modifying the chemical structure of these ASOs can enhance their targeting ability and reduce the side effects associated with the treatment.
Lead author Taiki Matsubayashi from TMDU explains, “We have recently developed a new chemical modification called BNAP-AEO. While ASOs carrying BNAP-AEO are anticipated to be highly effective, their biological efficacy and toxicity have yet to be investigated.”
To explore the potential of BNAP-AEO, the researchers conducted a series of experiments using cell cultures and animal models. Their findings revealed that the modified sugar significantly increased the therapeutic efficacy of the ASOs while reducing their toxic side effects.
These results demonstrate the potential of BNAP-AEO as a valuable modification for ASOs targeting CNS diseases. Further research is needed to fully understand the underlying mechanisms and optimize the use of this modification for clinical applications.
In summary, the researchers’ discovery of the enhanced safety and efficacy of ASOs carrying the BNAP-AEO modification holds great promise for the development of more effective treatments for CNS diseases.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it.
