UTRN and spinal muscular atrophy: In fact, many previous studies have reported that modified U7 snRNA enables the splicing modulation of various genes in vivo, such as in a Duchenne muscular dystrophy (DMD) mouse model (D2-mdx mice [4], mdx mice [9], mdx52 mice [16], utrophin/dystrophin double-knockout mice [13], Dmd exon 2 duplication model mice [34]), an amyotrophic lateral sclerosis (ALS) mouse model [35], and a spinal muscular atrophy (SMA) mouse model [36].