Abstract:
:Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models for DMD are genetically homologous and most commonly used in pre-clinical testing. Although the mouse is a strong, proof-of-concept model, affected dogs show more analogous clinical and immunological disease progression compared to boys with DMD. As such, evaluating genetic therapies in the canine models may better predict response at the genetic, phenotypic, and immunological levels. We review the use of canine models for DMD and their benefits as it pertains to genetic therapy studies, including gene replacement, exon skipping, and gene editing.
journal_name
Hum Genetjournal_title
Human geneticsauthors
Nghiem PP,Kornegay JNdoi
10.1007/s00439-019-01976-zsubject
Has Abstractpub_date
2019-05-01 00:00:00pages
483-489issue
5eissn
0340-6717issn
1432-1203pii
10.1007/s00439-019-01976-zjournal_volume
138pub_type
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