(Q34725351)

4 August 2017

Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy (English)

1 reference

4 August 2017

spinal muscular atrophy

1 reference

Darlise DiMatteo

1

1 reference

4 August 2017

Stephanie Callahan

series ordinal

2

1 reference

4 August 2017

Eric B Kmiec

series ordinal

3

1 reference

4 August 2017

language of work or name

English

0 references

publication date

24 October 2007

1 reference

Experimental Cell Research

4 August 2017

1 reference

4 August 2017

314

1 reference

4 August 2017

4

1 reference

4 August 2017

878-886

1 reference

Identification and characterization of a spinal muscular atrophy-determining gene

4 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Animal models of spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Development of a gene therapy strategy for the restoration of survival motor neuron protein expression: implications for spinal muscular atrophy therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Gene therapy progress and prospects: targeted gene repair

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Determination of SMN1 and SMN2 copy number using TaqMan technology

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Improved single-cell protocol for preimplantation genetic diagnosis of spinal muscular atrophy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Strand bias in targeted gene repair is influenced by transcriptional activity

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

DNA replication and transcription direct a DNA strand bias in the process of targeted gene repair in mammalian cells.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

The survival motor neuron protein in spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Reaction parameters of targeted gene repair in mammalian cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

A tolerance of DNA heterology in the mammalian targeted gene repair reaction

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Strand bias in oligonucleotide-mediated dystrophin gene editing

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Valproic acid increases SMN levels in spinal muscular atrophy patient cells.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Treatment of spinal muscular atrophy by sodium butyrate

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Establishing a standardized therapeutic testing protocol for spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

Gene-based treatment of motor neuron diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2007.10.012

10.1016/J.YEXCR.2007.10.012

7 January 2021

Identifiers

DOI

1 reference

4 August 2017

1 reference