Journal article
The emperor's new dystrophin: Finding sense in the noise
Trends in Molecular Medicine, Vol.21(7), pp.417-426
2015
Abstract
Targeted dystrophin exon removal is a promising therapy for Duchenne muscular dystrophy (DMD); however, dystrophin expression in some reports is not supported by the associated data. As in the account of ‘The Emperor's New Clothes’, the validity of such claims must be questioned, with critical re-evaluation of available data. Is it appropriate to report clinical benefit and induction of dystrophin as dose dependent when the baseline is unclear? The inability to induce meaningful levels of dystrophin does not mean that dystrophin expression as an end point is irrelevant, nor that induced exon skipping as a strategy is flawed, but demands that drug safety and efficacy, and study parameters be addressed, rather than questioning the strategy or the validity of dystrophin as a biomarker.
Details
- Title
- The emperor's new dystrophin: Finding sense in the noise
- Authors/Creators
- S.D. Wilton (Author/Creator)R.N. Veedu (Author/Creator)S. Fletcher (Author/Creator)
- Publication Details
- Trends in Molecular Medicine, Vol.21(7), pp.417-426
- Publisher
- Elsevier BV
- Identifiers
- 991005540798507891
- Copyright
- © 2015 Elsevier Ltd.
- Murdoch Affiliation
- Centre for Comparative Genomics
- Language
- English
- Resource Type
- Journal article
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Source: InCites
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- Citation topics
- 1 Clinical & Life Sciences
- 1.255 Musculoskeletal Disorders
- 1.255.628 Duchenne Muscular Dystrophy
- Web Of Science research areas
- Biochemistry & Molecular Biology
- Cell Biology
- Medicine, Research & Experimental
- ESI research areas
- Molecular Biology & Genetics