SMN2 and spinal muscular atrophy: Validated targets can modulate mRNA splicing (Palacino et al., 2015; Ratni et al., 2018) or modify the expression of factors implicated in human disease (Havens et al., 2013; Havens and Hastings, 2016; Montes et al., 2019; Hill and Meisler, 2021), such as inducing SMN2 expression in spinal muscular atrophy (Hua et al., 2008; Hua et al., 2010).