Figure 1. The potential RNA-targeted druggable genome.
Only a small fraction of the human genome has been successfully drugged to date. As shown in the pie chart, only ~1.5% of the genome encodes proteins (corresponding to ~20,000 proteins)1,2. As shown in the expanded pie segment above, an estimated 10–15% of proteins are thought to be disease-related3,4,5 (~2,000–3,000 proteins; encoded by 0.2% of the genome). Currently approved drugs interact therapeutically with <700 of these proteins (encoded by 0.05% of the human genome)6. Targeting RNAs could expand on the proportion of the human genome that could be therapeutically targeted. Possible RNA targets include mRNAs that encode disease-related proteins that have been characterized as undruggable or difficult to drug (shown in light blue in the pie segment) and also non-coding RNAs that influence disease (corresponding to an unknown proportion of the ~70% of the genome that encodes non-coding RNAs).