Journal
BIOMARKERS IN MEDICINE
Volume 13, Issue 14, Pages 1209-1225Publisher
FUTURE MEDICINE LTD
DOI: 10.2217/bmm-2019-0242
Keywords
Duchenne muscular dystrophy; dystrophin; exon-skipping; muscle; oligonucleotide drug; phosphorodiamidate morpholino oligomer; surrogate biomarker
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Funding
- NS Pharma Inc.
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Aim: Detection of drug-induced dystrophin in patient muscle biopsy is a surrogate outcome measure for Duchenne muscular dystrophy. We sought to establish and validate an orthogonal approach to measurement of dystrophin protein and RNA in muscle biopsies. Materials & methods: Validated methods were developed for dystrophin western blotting, mass spectrometry, immunostaining and reverse transcriptase PCR of biopsy mRNA using muscle biopsy standards. Results: Both western blotting and mass spectrometry validated methods demonstrated good linearity, and acceptable precision and accuracy with a lower limit of quantitation at 1%. Immunostaining and reverse transcriptase PCR methods were shown to be reliable. Conclusion: The described orthogonal approach is sufficient to support measures of dystrophin as a surrogate outcome in a clinical trial.
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