Gender-Specific Amelioration of SMA Phenotype upon Disruption of a Deep Intronic Structure by an Oligonucleotide

Autor: Howell, Matthew D., Ottesen, Eric W., Singh, Natalia N., Anderson, Rachel L., Singh, Ravindra N.
Zdroj: In Molecular Therapy 7 June 2017 25(6):1328-1341
Databáze: ScienceDirect