Autoimmune response and its long‐term consequences after exon‐skipping therapy in a Duchenne muscular dystrophy mouse model

The progress of antisense‐based therapies using first generation Morpholino oligonucleotides for Duchenne muscular dystrophy (DMD) is expected to partially restore dystrophin expression and may prolong the lifespan of DMD patients. In a recent issue of The Journal of Pathology, a sophisticated study...

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Bibliographic Details
Published in:The Journal of pathology 2019-11, Vol.249 (3), p.271-273
Main Authors: Nordin, Joel Z, Aoki, Yoshitsugu
Format: Article
Language:English
Subjects:
Animals
Antisense therapy
Autoimmunity
DMD
Duchenne muscular dystrophy
Duchenne's muscular dystrophy
Dystrophin
Dystrophin - genetics
Dystrophy
Exon skipping
Exons
Humans
humoral
immune response
Life span
Medicin och hälsovetenskap
Mice
Mice, Inbred mdx
Morpholino
Morpholinos
Muscles
Muscular dystrophy
Muscular Dystrophy, Duchenne - genetics
Oligonucleotides
Oligonucleotides, Antisense
Skeletal muscle
United Kingdom
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Summary:The progress of antisense‐based therapies using first generation Morpholino oligonucleotides for Duchenne muscular dystrophy (DMD) is expected to partially restore dystrophin expression and may prolong the lifespan of DMD patients. In a recent issue of The Journal of Pathology, a sophisticated study by Vila et al used a dystrophic mouse model of DMD to demonstrate that Morpholino‐induced exon skipping induced dystrophin expression in skeletal muscle and stimulated cell mediated and humoral responses to dystrophin. The study highlights the need to further investigate the autoimmune response against de novo synthesised truncated dystrophin protein and its long‐term consequences after exon‐skipping therapy for DMD. © 2019 Pathological Society of Great Britain and Ireland. Published by John Wiley & Sons, Ltd.
ISSN:0022-3417
1096-9896
1096-9896
DOI:10.1002/path.5327