Research graph
References from Utargetome: A targetome prediction tool for modified U1-snRNAs to identify distal-target positions with improved selectivity. Local targets link to admitted publications; unresolved targets remain external evidence.
Insights into the U1 small nuclear ribonucleoprotein complex superfamily
10.1002/wrna.1257 · 2015 · External reference
RNA Splicing by the Spliceosome
10.1146/annurev-biochem-091719-064225 · 2020 · External reference
U1 snRNP Telescripting: Suppression of Premature Transcription Termination in Introns as a New Layer of Gene Regulation
10.1101/cshperspect.a032235 · 2019 · External reference
Gene therapy with modified U1 small nuclear RNA
10.1080/17446651.2017.1316191 · 2017 · External reference
RNA mis-splicing in disease
10.1038/nrg.2015.3 · 2016 · External reference
Splicing mutations in human genetic disorders: examples, detection, and confirmation
10.1007/s13353-018-0444-7 · 2018 · External reference
Noncanonical registers and base pairs in human 5′ splice-site selection
10.1093/nar/gkw163 · 2016 · External reference
Pick one, but be quick: 5′ splice sites and the problems of too many choices
10.1101/gad.209759.112 · 2013 · External reference
Widespread recognition of 5′ splice sites by noncanonical base-pairing to U1 snRNA involving bulged nucleotides
10.1101/gad.190173.112 · 2012 · External reference
A novel approach to describe a U1 snRNA binding site
10.1093/nar/gkg901 · 2003 · External reference
Extended base pair complementarity between U1 snRNA and the 5′ splice site does not inhibit splicing in higher eukaryotes, but rather increases 5′ splice site recognition
10.1093/nar/gki824 · 2005 · External reference
Determinants of the inherent strength of human 5′ splice sites
10.1261/rna.2040605 · 2005 · External reference
Modulating role of RNA structure in alternative splicing of a critical exon in the spinal muscular atrophy genes
10.1093/nar/gkl1050 · 2007 · External reference
An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects
10.1093/hmg/dds045 · 2012 · External reference
Activation of a cryptic 5′ splice site reverses the impact of pathogenic splice site mutations in the spinal muscular atrophy gene
10.1093/nar/gkx824 · 2017 · External reference
Small nuclear RNA-mediated modulation of splicing reveals a therapeutic strategy for a TREM2 mutation and its post-transcriptional regulation
10.1038/s41598-018-25204-2 · 2018 · External reference
Perspective in Alternative Splicing Coupled to Nonsense-Mediated mRNA Decay
10.3390/ijms21249424 · 2020 · External reference
A new type of mutation causes a splicing defect in ATM
10.1038/ng858 · 2002 · External reference
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model
10.1073/pnas.0508917103 · 2006 · External reference
Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639 + 861C>T and c.639 + 919G>A GLA Deep Intronic Mutations
2016 · External reference
Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator (RPGR) Splice Defect
10.1089/nat.2021.0053 · 2022 · External reference
Inhibiting expression of specific genes in mammalian cells with 5′ end-mutated U1 small nuclear RNAs targeted to terminal exons of pre-mRNA
10.1073/pnas.1332669100 · 2003 · External reference
Requirements for gene silencing mediated by U1 snRNA binding to a target sequence
10.1093/nar/gkn068 · 2008 · External reference
Gene silencing by synthetic U1 adaptors
10.1038/nbt.1525 · 2009 · External reference
Therapeutic activity of modified U1 core spliceosomal particles
10.1038/ncomms11168 · 2016 · External reference
A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I
10.3390/ijms21062136 · 2020 · External reference
Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA
10.1093/nar/gkz469 · 2019 · External reference
A U1i RNA that Enhances HIV-1 RNA Splicing with an Elongated Recognition Domain Is an Optimal Candidate for Combination HIV-1 Gene Therapy
10.1016/j.omtn.2019.10.011 · 2019 · External reference
Large-scale comparative analysis of splicing signals and their corresponding splicing factors in eukaryotes
10.1101/gr.6818908 · 2008 · External reference
Detection and removal of biases in the analysis of next-generation sequencing reads
10.1371/journal.pone.0016685 · 2011 · External reference
Profiling of cis- and trans-acting factors supporting noncanonical splice site activation
10.1080/15476286.2020.1798111 · 2021 · External reference
Recognition of atypical 5′ splice sites by shifted base-pairing to U1 snRNA
10.1038/nsmb.1546 · 2009 · External reference
RNA-RNA interactions enable specific targeting of noncoding RNAs to nascent Pre-mRNAs and chromatin sites
10.1016/j.cell.2014.08.018 · 2014 · External reference
The conserved AU dinucleotide at the 5′ end of nascent U1 snRNA is optimized for the interaction with nuclear cap-binding-complex
10.1093/nar/gkx608 · 2017 · External reference
U1-snRNA-mediated rescue of mRNA processing in severe factor VII deficiency
10.1182/blood-2007-10-117440 · 2008 · External reference
An Altered Splicing Registry Explains the Differential ExSpeU1-Mediated Rescue of Splicing Mutations Causing Haemophilia A
10.3389/fgene.2019.00974 · 2019 · External reference
A novel role of U1 snRNP: Splice site selection from a distance
10.1016/j.bbagrm.2019.04.004 · 2019 · External reference
Therapeutic strategy to rescue mutation-induced exon skipping in rhodopsin by adaptation of U1 snRNA
10.1002/humu.20861 · 2009 · External reference
Multiple Isoforms of Nesprin1 Are Integral Components of Ciliary Rootlets
10.1016/j.cub.2017.05.066 · 2017 · External reference
Ca2+ sensor synaptotagmin-1 mediates exocytosis in mammalian photoreceptors
10.7554/elife.45946 · 2019 · External reference
An Overview of the Genetics of ABCA4 Retinopathies, an Evolving Story
10.3390/genes12081241 · 2021 · External reference
Mutations in CEP78 Cause Cone-Rod Dystrophy and Hearing Loss Associated with Primary-Cilia Defects
10.1016/j.ajhg.2016.07.009 · 2016 · External reference
Mutations within the cGMP-binding domain of CNGA1 causing autosomal recessive retinitis pigmentosa in human and animal model
10.1038/s41420-022-01185-0 · 2022 · External reference
Retinal Structure and Function in a Knock-in Mouse Model for the FAM161A-p.Arg523 * Human Nonsense Pathogenic Variant
10.1016/j.xops.2022.100229 · 2023 · External reference
Retinal Dystrophy Associated with Homozygous Variants in NRL
10.3390/genes15121594 · 2024 · External reference
PRPF4 mutations cause autosomal dominant retinitis pigmentosa
10.1093/hmg/ddu005 · 2014 · External reference
RDH5 and RLBP1-Associated Inherited Retinal Diseases: Refining the Spectrum of Stationary and Progressive Phenotypes
10.1016/j.ajo.2024.06.016 · 2024 · External reference
Retinal Dystrophy Associated With RLBP1 Retinitis Pigmentosa: A Five-Year Prospective Natural History Study
10.1167/iovs.64.13.42 · 2023 · External reference
Retinal Degeneration Associated With RPGRIP1: A Review of Natural History, Mutation Spectrum, and Genotype-Phenotype Correlation in 228 Patients
10.3389/fcell.2021.746781 · 2021 · External reference
Rescue of coagulation factor VII function by the U1 + 5A snRNA
10.1182/blood-2009-03-207613 · 2009 · External reference
Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model
10.1093/hmg/ddy151 · 2018 · External reference
Efficient use of a “dead-end” GA 5′ splice site in the human fibroblast growth factor receptor genes
10.1093/emboj/cdg163 · 2003 · External reference
Absolute quantitative and base-resolution sequencing reveals comprehensive landscape of pseudouridine across the human transcriptome
10.1038/s41592-024-02439-8 · 2024 · External reference
The contribution of pseudouridine to stabilities and structure of RNAs
10.1093/nar/gkt1330 · 2014 · External reference
Pseudouridines in RNAs: switching atoms means shifting paradigms
10.1002/1873-3468.14188 · 2021 · External reference
Pseudouridine synthases modify human pre-mRNA co-transcriptionally and affect pre-mRNA processing
10.1016/j.molcel.2021.12.023 · 2022 · External reference
Basic local alignment search tool
10.1016/s0022-2836(05)80360-2 · 1990 · External reference
A computational analysis of antisense off-targets in prokaryotic organisms
10.1016/j.ygeno.2014.11.010 · 2015 · External reference
ViennaRNA Package 2.0
10.1186/1748-7188-6-26 · 2011 · External reference
True genes for human U1 small nuclear RNA. Copy number, polymorphism, and methylation
10.1016/s0021-9258(17)43509-5 · 1984 · External reference
Inflammatory adipose activates a nutritional immunity pathway leading to retinal dysfunction
10.1016/j.celrep.2022.110942 · 2022 · External reference
A simplified technique for in situ excision of cornea and evisceration of retinal tissue from human ocular globe
10.3791/3765-v · 2012 · External reference
In-Depth Molecular Profiling Specifies Human Retinal Microglia Identity
10.3389/fimmu.2022.863158 · 2022 · External reference
Human retinal organoids release extracellular vesicles that regulate gene expression in target human retinal progenitor cells
10.1038/s41598-021-00542-w · 2021 · External reference
Twelve years of SAMtools and BCFtools
10.1093/gigascience/giab008 · 2021 · External reference
Graph-based genome alignment and genotyping with HISAT2 and HISAT-genotype
10.1038/s41587-019-0201-4 · 2019 · External reference
StringTie enables improved reconstruction of a transcriptome from RNA-seq reads
10.1038/nbt.3122 · 2015 · External reference
DAVID: a web server for functional enrichment analysis and functional annotation of gene lists (2021 update)
10.1093/nar/gkac194 · 2022 · External reference
