Journal article icon

Journal article : Review

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

Abstract:

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphorodiamidate morpholino backbone (PMO) of ASOs generated the peptide-conjugated PMOs (PPMOs) that exhibit a dramatically improved pharmacokinetic profile. When tested in animal models, PPMOs demonstr...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Files:
Publisher copy:
10.1016/j.ebiom.2019.06.036

Authors


More by this author
Role:
Author
ORCID:
0000-0003-3610-3469
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Paediatrics
Role:
Author
ORCID:
0000-0002-5436-6011
Expand authors...
Publisher:
Elsevier Publisher's website
Journal:
EBioMedicine Journal website
Volume:
45
Pages:
630-645
Publication date:
2019-06-27
Acceptance date:
2019-06-18
DOI:
EISSN:
2352-3964
Pmid:
31257147
Language:
English
Keywords:
Subtype:
Review
Pubs id:
1027006
Local pid:
pubs:1027006
Deposit date:
2021-06-25

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP