Nicholas Owen
Nicholas Owen
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Automatic
Spinal Muscular Atrophy
Design principles for bifunctional targeted oligonucleotide enhancers of splicing
Controlling the patterns of splicing of specific genes is an important goal in the development of new therapies. We have shown that the splicing of a refractory exon, SMN2 exon 7, could be increased in fibroblasts derived from patients with spinal muscular atrophy by using bifunctional targeted oligonucleotide enhancers of splicing (TOES) oligonucleotides …
Nicholas Owen
,
H Zhou
,
A Malygin
,
F Muntoni
,
I Eperon
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Characterization of the Schizosaccharomyces pombe orthologue of the human survival motor neuron (SMN) protein
Childhood onset spinal muscular atrophy (SMA) is a common autosomal recessive disorder primarily characterized by the loss of lower alpha motor neurons. The underlying chromosomal defects causing SMA have been found in the survival motor neuron (SMN) gene. SMN has been ..
Nicholas Owen
,
CL Doe
,
J Mellor
,
KE Davies
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