Title |
Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching
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Published in |
BMC Medical Genomics, October 2011
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DOI | 10.1186/1471-2350-12-141 |
Pubmed ID | |
Authors |
Clayton T Fragall, Abbie M Adams, Russell D Johnsen, Ryszard Kole, Sue Fletcher, Steve D Wilton |
Abstract |
Antisense oligomer induced exon skipping aims to reduce the severity of Duchenne muscular dystrophy by redirecting splicing during pre-RNA processing such that the causative mutation is by-passed and a shorter but partially functional Becker muscular dystrophy-like dystrophin isoform is produced. Normal exons are generally targeted to restore the dystrophin reading frame however, an appreciable subset of dystrophin mutations are intra-exonic and therefore have the potential to compromise oligomer efficiency, necessitating personalised oligomer design for some patients. Although antisense oligomers are easily personalised, it remains unclear whether all patient polymorphisms within antisense oligomer target sequences will require the costly process of producing and validating patient specific compounds. |
X Demographics
Geographical breakdown
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Unknown | 2 | 100% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Korea, Republic of | 1 | 4% |
Unknown | 22 | 96% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 6 | 26% |
Student > Ph. D. Student | 3 | 13% |
Student > Postgraduate | 2 | 9% |
Professor > Associate Professor | 2 | 9% |
Student > Master | 2 | 9% |
Other | 4 | 17% |
Unknown | 4 | 17% |
Readers by discipline | Count | As % |
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Agricultural and Biological Sciences | 7 | 30% |
Medicine and Dentistry | 5 | 22% |
Biochemistry, Genetics and Molecular Biology | 4 | 17% |
Unspecified | 2 | 9% |
Social Sciences | 1 | 4% |
Other | 0 | 0% |
Unknown | 4 | 17% |