Short duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy
Anne Rietz, Kevin J. Hodgetts, Hrvoje Lusic, Kevin M. Quist, Erkan Y. Osman, Christian L. Lorson, Elliot J. Androphy
The paper describes a splicing modifier compound that creates a tunable mouse model of spinal muscular atrophy, showing efficacy in preventing and reversing SMA phenotypes with short-term treatment.
Life Science Alliance · Nov 24, 2020Peer-reviewedNeuroscience