Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy Molecular Therapy 24, 1592 (september 2016). doi:10.1038/mt.2016.145 Authors: Erkan Y Osman, Charles W Washington, Kevin A Kaifer, Chiara Mazzasette, Teresa N Patitucci, Kyra M Florea, Madeline E Simon, Chien-Ping Ko, Allison D Ebert & Christian L Lorson
Source: Molecular Therapy - Category: Genetics & Stem Cells Authors: Source Type: research