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MSC Mechanisms Improve ASO Distribution in HD

Antisense oligonucleotides (ASOs) represent promising therapeutic strategies for the suppression of toxic protein production in neurogenetic diseases, especially in Huntington's disease (HD); however, the pattern and mechanism of distribution of ASOs in the brain remain unclear. In a new STEM CELLS study, researchers from the lab of Zhong Pei (Sun Yat-sen University, Guangzhou, Guangdong, China) revealed a significant role for the glymphatic system in ASO distribution and, additionally, they discovered that the intravenous injection of mesenchymal stem cells (MSCs) rescues the loss of AQP‐4 in an HD mouse model. Given the status of AQP‐4 as a critical channel in the brain glymphatic system, the authors found more ASOs in brain tissue and a lower mutant HTT level in an HD mouse model following MSC injection. Overall, Wu et al. propose MSC therapy to enhance ASO distribution and therapeutic outcomes in HD patients.