Gene therapies in canine models for Duchenne muscular dystrophy. Academic Article

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.

authors

• Nghiem, Peter

published proceedings

• Hum Genet

altmetric score

• 0.25

author list (cited authors)

• Nghiem, P. P., & Kornegay, J. N.

citation count

• 18

complete list of authors

• Nghiem, Peter P||Kornegay, Joe N

publication date

• May 2019

publisher

• Springer Nature  Publisher

published in

• Human Genetics  Journal

keywords

• Animals
• Disease Models, Animal
• Dogs
• Dystrophin
• Gene Editing
• Genetic Therapy
• Humans
• Male
• Mice
• Muscular Dystrophy, Duchenne

PubMed Central ID

• 30734120

Digital Object Identifier (DOI)

• 10.1007/s00439-019-01976-z

start page

• 483

end page

• 489

volume

• 138

issue

• 5

URL

• http://dx.doi.org/10.1007/s00439-019-01976-z