In addition, an ASO-based allele-specific mRNA knockdown/degradation approach for dominantly inherited RP caused by pathogenic variants in RHO is underway in a clinical trial (NCT04123626), as are two different approved treatments for spinal muscular atrophy and Duchenne muscular dystrophy based on ASOs that modulate the splicing process.98 This evidence concerns the gene RHO and spinal muscular atrophy.