Kuivaniemi H, Kontusaari S, Tromp G, Zhao M J, Sabol C, Prockop D J
Department of Biochemistry and Molecular Biology, Jefferson Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania 19107.
J Biol Chem. 1990 Jul 15;265(20):12067-74.
Identical G+1 mutations in three different introns of the gene for type III procollagen (COL3A1) that cause aberrant splicing of RNA were found in three probands with life-threatening variants of Ehlers-Danlos syndrome. Because the three mutations were in a gene with multiple and homologous exons, they provided an interesting test for factors that influence aberrant splicing. The G+1 to A mutation in intron 16 caused extensive exon skipping, the G+1 to A mutation in intron 20 caused both use of a cryptic splice site and retention of all the intron sequences, and the G+1 to A mutation in intron 42 caused efficient use of a single cryptic splice site. The different patterns of RNA splicing were not explained by evaluation of potential cryptic splice sites in the introns by either their homology with 5'-splice sites from other genes or by their delta G(0)37 values for binding to U1 RNA. Instead, the results suggested that the patterns of aberrant RNA splicing were primarily determined by the relative rates at which adjacent introns were normally spliced.
在三名患有危及生命的埃勒斯-当洛综合征变体的先证者中,发现III型前胶原(COL3A1)基因的三个不同内含子中存在相同的G+1突变,这些突变导致RNA的异常剪接。由于这三个突变位于一个具有多个同源外显子的基因中,它们为影响异常剪接的因素提供了一个有趣的测试。内含子16中的G+1到A突变导致广泛的外显子跳跃,内含子20中的G+1到A突变导致一个隐蔽剪接位点的使用以及所有内含子序列的保留,内含子42中的G+1到A突变导致一个单一隐蔽剪接位点的有效使用。通过评估内含子中潜在的隐蔽剪接位点与其他基因的5'-剪接位点的同源性或其与U1 RNA结合的ΔG(0)37值,无法解释不同的RNA剪接模式。相反,结果表明异常RNA剪接模式主要由相邻内含子正常剪接的相对速率决定。