RNA mis-splicing in disease

MM Scotti, MS Swanson - Nature Reviews Genetics, 2016 - nature.com
The human transcriptome is composed of a vast RNA population that undergoes further
diversification by splicing. Detecting specific splice sites in this large sequence pool is the …

Deep intronic mutations and human disease

R Vaz-Drago, N Custódio, M Carmo-Fonseca - Human genetics, 2017 - Springer
Next-generation sequencing has revolutionized clinical diagnostic testing. Yet, for a
substantial proportion of patients, sequence information restricted to exons and exon–intron …

A day in the life of the spliceosome

AG Matera, Z Wang - Nature reviews Molecular cell biology, 2014 - nature.com
One of the most amazing findings in molecular biology was the discovery that eukaryotic
genes are discontinuous, with coding DNA being interrupted by stretches of non-coding …

Frequent pathway mutations of splicing machinery in myelodysplasia

K Yoshida, M Sanada, Y Shiraishi, D Nowak, Y Nagata… - Nature, 2011 - nature.com
Myelodysplastic syndromes and related disorders (myelodysplasia) are a heterogeneous
group of myeloid neoplasms showing deregulated blood cell production with evidence of …

Lessons from non-canonical splicing

CR Sibley, L Blazquez, J Ule - Nature Reviews Genetics, 2016 - nature.com
Recent improvements in experimental and computational techniques that are used to study
the transcriptome have enabled an unprecedented view of RNA processing, revealing many …

Children born small for gestational age: differential diagnosis, molecular genetic evaluation, and implications

MJJ Finken, M van der Steen, CCJ Smeets… - Endocrine …, 2018 - academic.oup.com
Children born small for gestational age (SGA), defined as a birth weight and/or length
below− 2 SD score (SDS), comprise a heterogeneous group. The causes of SGA are …

The significant other: splicing by the minor spliceosome

JJ Turunen, EH Niemelä, B Verma… - Wiley Interdisciplinary …, 2013 - Wiley Online Library
The removal of non‐coding sequences, introns, from the mRNA precursors is an essential
step in eukaryotic gene expression. U12‐type introns are a minor subgroup of introns …

Pre-mRNA splicing in disease and therapeutics

RK Singh, TA Cooper - Trends in molecular medicine, 2012 - cell.com
In metazoans, alternative splicing of genes is essential for regulating gene expression and
contributing to functional complexity. Computational predictions, comparative genomics, and …

The spliceosome as a target of novel antitumour drugs

S Bonnal, L Vigevani, J Valcárcel - Nature reviews Drug discovery, 2012 - nature.com
Several bacterial fermentation products and their synthetic derivatives display antitumour
activities and bind tightly to components of the spliceosome, which is the complex molecular …

Compound inheritance of a low-frequency regulatory SNP and a rare null mutation in exon-junction complex subunit RBM8A causes TAR syndrome

CA Albers, DS Paul, H Schulze, K Freson… - Nature …, 2012 - nature.com
The exon-junction complex (EJC) performs essential RNA processing tasks 1, 2, 3, 4, 5.
Here, we describe the first human disorder, thrombocytopenia with absent radii (TAR) 6 …