Comparison of in silico strategies to prioritize rare genomic variants
impacting RNA splicing for the diagnosis of genomic disorders
Abstract
The development of computational methods to assess pathogenicity of
pre-messenger RNA splicing variants is critical for diagnosis of human
disease. We assessed the capability of eight algorithms, and a consensus
approach, to prioritize 250 variants of uncertain significance (VUS)
that underwent splicing functional analyses. It is the capability of
algorithms to differentiate VUSs away from the immediate splice site as
‘pathogenic’ or ‘benign’ that is likely to have the most substantial
impact on diagnostic testing. We show that SpliceAI is the best single
strategy in this regard, but that combined usage of tools using a
weighted approach can increase accuracy further. We incorporated
prioritization strategies alongside diagnostic testing for rare
disorders. We show that 15% of 2783 referred individuals carry rare
variants expected to impact splicing that were not initially identified
as ‘pathogenic’ or ‘likely pathogenic’; 1 in 5 of these cases could lead
to new or refined diagnoses.