Mesenchymal stem cells alleviate AQP ‐4‐dependent glymphatic dysfunction and improve brain distribution of antisense oligonucleotides in BACHD mice

In this study, the glymphatic system was found to be pivotal in ASO distribution. Moreover, an intravenously injection of MSCs (IV ‐MSC) rescued the loss of AQP‐4 in an HD mouse model. Since AQP‐4 is a critical channel in the brain glymphatic system, more ASOs were found in the brain tissue and a lower mutant HTT level was present in an HD mouse model with an IV‐MSC injection. In summary, IV‐MSC may be a potential the rapy to enhance ASO distribution efficiency and promote its therapeutic outcome in HD patients.

Source: Stem Cells - October 23, 2019 Category: Stem Cells Authors: Teng ‐teng Wu, Feng‐juan Su, Yan‐qing Feng, Bin Liu, Ming‐yue Li, Feng‐yin Liang, Ge Li, Xue‐jiao Li, Yu Zhang, Zhong‐qiong Cai, Zhong Pei Tags: Regenerative Medicine Source Type: research