Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation (Q28300972)

10

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author name string

Kathrin Meyer

1

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Julien Marquis

2

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Judith Trüb

3

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Rachel Nlend Nlend

4

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Sonia Verp

5

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Hans Imboden

7

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Isabelle Barde

8

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Didier Trono

9

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Daniel Schümperli

The regulation and regulatory activities of alternative splicing of the SMN gene

10

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language of work or name

English

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publication date

1 February 2009

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published in

Human Molecular Genetics

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volume

18

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issue

3

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page(s)

546-55

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cites work

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Spinal muscular atrophy: from gene to therapy

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Identification and characterization of a spinal muscular atrophy-determining gene

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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

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Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy

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Spinal muscular atrophy: position and functional importance of the branch site preceding SMN exon 7.

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Evolving concepts on human SMN pre-mRNA splicing.

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

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Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.

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

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Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

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

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

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Spinal muscular atrophy: SMN2 pre-mRNA splicing corrected by a U7 snRNA derivative carrying a splicing enhancer sequence

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The survival motor neuron protein in spinal muscular atrophy

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Genotypic features of lentivirus transgenic mice

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The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy

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Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

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Intramuscular injection of AAV-GDNF results in sustained expression of transgenic GDNF, and its delivery to spinal motoneurons by retrograde transport

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Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons

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Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1.

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Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs

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SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing

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Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

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Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector

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Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos

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