
Proteins PRPF31, PRPF3 and PRPF8 (RP-PRPFs) are ubiquitously expressed components of the spliceosome, a macromolecular complex that processes nearly all pre-mRNAs. Although these spliceosomal proteins are conserved in eukaryotes and are essential for survival, heterozygous mutations in human RP-PRPF genes lead to retinitis pigmentosa, a hereditary disease restricted to the eye. Using cells from patients with 10 different mutations, we show that all clinically relevant RP-PRPF defects affect the stoichiometry of spliceosomal small nuclear RNAs (snRNAs), the protein composition of tri-small nuclear ribonucleoproteins and the kinetics of spliceosome assembly. These mutations cause inefficient splicing in vitro and affect constitutive splicing ex-vivo by impairing the removal of at least 9% of endogenously expressed introns. Alternative splicing choices are also affected when RP-PRPF defects are present. Furthermore, we show that the steady-state levels of snRNAs and processed pre-mRNAs are highest in the retina, indicating a particularly elevated splicing activity. Our results suggest a role for PRPFs defects in the etiology of PRPF-linked retinitis pigmentosa, which appears to be a truly systemic splicing disease. Although these mutations cause widespread and important splicing defects, they are likely tolerated by the majority of human tissues but are critical for retinal cell survival.
Male, Heterozygote, Ribonucleoprotein, U4-U6 Small Nuclear, RNA Splicing, Nuclear Proteins, RNA-Binding Proteins, Ribonucleoproteins, Small Nuclear, Introns, Retina, Alternative Splicing, Cell Line, Tumor, RNA, Small Nuclear, Mutation, RNA Precursors, Humans, Female, Carrier Proteins, Eye Proteins, Retinitis Pigmentosa, Genes, Dominant
Male, Heterozygote, Ribonucleoprotein, U4-U6 Small Nuclear, RNA Splicing, Nuclear Proteins, RNA-Binding Proteins, Ribonucleoproteins, Small Nuclear, Introns, Retina, Alternative Splicing, Cell Line, Tumor, RNA, Small Nuclear, Mutation, RNA Precursors, Humans, Female, Carrier Proteins, Eye Proteins, Retinitis Pigmentosa, Genes, Dominant
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