(Q34004566)

30 July 2017

title

Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing (English)

1 reference

30 July 2017

Gregory E Tullis

0 references

Tristan H Coady

1 reference

30 July 2017

Monir Shababi

series ordinal

1 reference

30 July 2017

Christian L Lorson

series ordinal

1 reference

30 July 2017

publication date

5 June 2007

1 reference

30 July 2017

1 reference

30 July 2017

1 reference

30 July 2017

1 reference

30 July 2017

page(s)

1471-1478

1 reference

The neurobiology of childhood spinal muscular atrophy.

30 July 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Identification and characterization of a spinal muscular atrophy-determining gene

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Survival motor neuron gene transcript analysis in muscles from spinal muscular atrophy patients.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Therapeutics development for spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2)

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

hnRNP-G promotes exon 7 inclusion of survival motor neuron (SMN) via direct interaction with Htra2-beta1

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

In Vitro Restoration of Functional SMN Protein in Human Trophoblast Cells Affected by Spinal Muscular Atrophy by Small Fragment Homologous Replacement

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Correction of disease-associated exon skipping by synthetic exon-specific activators

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Alternative splicing in disease and therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

SMN oligomerization defect correlates with spinal muscular atrophy severity

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

The exon 2b region of the spinal muscular atrophy protein, SMN, is involved in self-association and SIP1 binding

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

The survival motor neuron protein in spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Why do cells need an assembly machine for RNA-protein complexes?

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

5' exon replacement and repair by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Spliceosome-mediated RNA trans-splicing as a tool for gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A novel peptide defined through phage display for therapeutic protein and vector neuronal targeting

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

AAV hybrid serotypes: improved vectors for gene delivery

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Adeno-associated virus vectors can be efficiently produced without helper virus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Gemin proteins are required for efficient assembly of Sm-class ribonucleoproteins.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300222

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300222

1 reference

30 July 2017

1 reference