| scholarly article | Q13442814 |
| P2093 | author name string | Klemens J Hertel | |
| Bianca J Lam | |||
| Till Geib | |||
| Sharlene R Lim | |||
| Csilla Madocsai | |||
| P2860 | cites work | Identification and characterization of Gemin7, a novel component of the survival of motor neuron complex | Q24299793 |
| hnRNP-G promotes exon 7 inclusion of survival motor neuron (SMN) via direct interaction with Htra2-beta1 | Q24303882 | ||
| The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins | Q24316085 | ||
| A novel nuclear structure containing the survival of motor neurons protein | Q24324247 | ||
| Sorting out the complexity of SR protein functions | Q24539813 | ||
| Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs | Q24569504 | ||
| Assembly, nuclear import and function of U7 snRNPs studied by microinjection of synthetic U7 RNA into Xenopus oocytes | Q24624098 | ||
| Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human beta-thalassemic mutations | Q28137724 | ||
| SMN, the product of the spinal muscular atrophy gene, binds preferentially to dimethylarginine-containing protein targets | Q28199238 | ||
| Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy | Q28201294 | ||
| SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1 | Q28204721 | ||
| Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients | Q28209134 | ||
| The SMN complex | Q28259557 | ||
| The special Sm core structure of the U7 snRNP: far-reaching significance of a small nuclear ribonucleoprotein | Q28291688 | ||
| Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping | Q28291830 | ||
| Formation of the 3' end of histone mRNA | Q28610112 | ||
| Identification and characterization of a spinal muscular atrophy-determining gene | Q29547495 | ||
| A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy | Q29617367 | ||
| Improved free-energy parameters for predictions of RNA duplex stability | Q29618274 | ||
| The gene frequency of acute Werdnig-Hoffmann disease (SMA type 1). A total population survey in North-East England | Q33585740 | ||
| Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular atrophy | Q33588368 | ||
| Exonic splicing enhancers: mechanism of action, diversity and role in human genetic diseases | Q33850031 | ||
| A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. | Q33864982 | ||
| Treatment of spinal muscular atrophy by sodium butyrate | Q33933841 | ||
| A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy. | Q34101383 | ||
| Structure and organization of the human survival motor neurone (SMN) gene | Q34400462 | ||
| Essential role for the SMN complex in the specificity of snRNP assembly | Q34529450 | ||
| Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts | Q34921907 | ||
| Antisense-mediated redirection of mRNA splicing | Q35162961 | ||
| Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2) | Q35210565 | ||
| snRNAs contain specific SMN-binding domains that are essential for snRNP assembly | Q37012661 | ||
| Implications of ribozyme kinetics for targeting the cleavage of specific RNA molecules in vivo: more isn't always better | Q37571777 | ||
| Identification of a cis-acting element for the regulation of SMN exon 7 splicing | Q38290136 | ||
| Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing | Q38296244 | ||
| The role of histone acetylation in SMN gene expression. | Q40437533 | ||
| Valproic acid increases SMN levels in spinal muscular atrophy patient cells. | Q40620622 | ||
| Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1. | Q40741164 | ||
| Correlation between severity and SMN protein level in spinal muscular atrophy | Q41102726 | ||
| The genetic component in child mortality | Q42041759 | ||
| Correction of disease-associated exon skipping by synthetic exon-specific activators | Q44276105 | ||
| Active transport of the survival motor neuron protein and the role of exon-7 in cytoplasmic localization. | Q48586550 | ||
| Deletions in the survival motor neuron gene on 5q13 in autosomal recessive spinal muscular atrophy. | Q52508889 | ||
| Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. | Q55035683 | ||
| DNA cloning without restriction enzyme and ligase | Q73345625 | ||
| A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy | Q73588971 | ||
| SMN oligomerization defect correlates with spinal muscular atrophy severity | Q74557706 | ||
| P433 | issue | 6 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 1013-22 | |
| P577 | publication date | 2005-12-01 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs | |
| P478 | volume | 12 |
| Q34132150 | A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo |
| Q38300319 | A sensitive assay for measuring SMN mRNA levels in peripheral blood and in muscle samples of patients affected with spinal muscular atrophy |
| Q28244480 | A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy |
| Q35180737 | Absence of an intron splicing silencer in porcine Smn1 intron 7 confers immunity to the exon skipping mutation in human SMN2. |
| Q42156108 | An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects |
| Q34336279 | Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy |
| Q34030144 | Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model |
| Q26864605 | Applicability of histone deacetylase inhibition for the treatment of spinal muscular atrophy |
| Q35699768 | Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides |
| Q37154271 | Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy |
| Q21092173 | Development of a single vector system that enhances trans-splicing of SMN2 transcripts |
| Q24651863 | Differential 3' splice site recognition of SMN1 and SMN2 transcripts by U2AF and U2 snRNP |
| Q35683524 | Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA. |
| Q28290763 | Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes |
| Q37696051 | Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene |
| Q51826765 | Emerging treatment options for spinal muscular atrophy |
| Q33278100 | Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon |
| Q33722582 | Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA. |
| Q31002975 | Genetically modified pig models for neurodegenerative disorders |
| Q34902920 | Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA. |
| Q33241167 | Linking splicing to Pol II transcription stabilizes pre-mRNAs and influences splicing patterns |
| Q36322222 | Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy |
| Q36866549 | Modulating the expression of disease genes with RNA-based therapy. |
| Q27007782 | Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA) |
| Q42218490 | Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models |
| Q37255013 | Novel therapeutic modalities to address nondrugable protein interaction targets |
| Q37609948 | Progress in therapeutic antisense applications for neuromuscular disorders |
| Q27489807 | Progress toward therapy with antisense-mediated splicing modulation |
| Q29615183 | RNA and disease |
| Q42367284 | RNA splicing process analysis for identifying antisense oligonucleotide inhibitors with padlock probe-based isothermal amplification |
| Q34762623 | Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy. |
| Q34004566 | Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing |
| Q21142695 | Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs |
| Q29013847 | SMN in spinal muscular atrophy and snRNP biogenesis |
| Q34312156 | SMN-Inducing Compounds For The Treatment Of Spinal Muscular Atrophy |
| Q34460251 | Spinal Muscular Atrophy Therapeutics: Where do we Stand? |
| Q21202863 | Spinal muscular atrophy |
| Q37033848 | Spinal muscular atrophy and a model for survival of motor neuron protein function in axonal ribonucleoprotein complexes |
| Q37714861 | Spinal muscular atrophy disease: a literature review for therapeutic strategies |
| Q28302547 | Spinal muscular atrophy: SMN2 pre-mRNA splicing corrected by a U7 snRNA derivative carrying a splicing enhancer sequence |
| Q33871311 | Spinal muscular atrophy: mechanisms and therapeutic strategies |
| Q34065993 | Splicing of the Survival Motor Neuron genes and implications for treatment of SMA |
| Q37426472 | Splicing therapeutics in SMN2 and APOB. |
| Q27009581 | Splicing therapy for neuromuscular disease |
| Q34664690 | State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications. |
| Q54610896 | Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. |
| Q24648050 | The RNA binding protein hnRNP Q modulates the utilization of exon 7 in the survival motor neuron 2 (SMN2) gene |
| Q35794628 | The Silent Sway of Splicing by Synonymous Substitutions |
| Q36768787 | Therapeutic activity of modified U1 core spliceosomal particles |
| Q33720730 | Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy |
| Q28248354 | U7 snRNA-mediated correction of aberrant splicing caused by activation of cryptic splice sites |
| Q38289261 | Viral Vector-Mediated Antisense Therapy for Genetic Diseases |