NeuroscienceLife Science AlliancePeer-reviewed
Short duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy
Anne Rietz, Kevin J. Hodgetts, Hrvoje Lusic…·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.
2 citations