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Accession IconSRP067305

Mus musculus RNA-SEQ raw sequence reads

Organism Icon Mus musculus
Sample Icon 6 Downloadable Samples
Technology Badge IconIllumina HiSeq 2500

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Description
We evaluated the therapeutic activity of the modified U1 particles in a mouse model affected by severe spinal muscular atrophy. ExSpeU1 introduced by germline transgenesis efficiently rescued the phenotype increasing SMN2 exon 7 splicing, SMN protein production and radically extending the life span.
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