Journal
DISEASE MODELS & MECHANISMS
Volume 13, Issue 2, Pages -Publisher
COMPANY BIOLOGISTS LTD
DOI: 10.1242/dmm.041673
Keywords
Genetic therapies; Genome editing; Exon skipping; Pre-clinical studies; Muscular dystrophy
Categories
Funding
- Duchenne Parent Project
Ask authors/readers for more resources
For many genetic diseases, researchers are developing personalized medicine approaches. These sometimes employ custom genetic interventions such as antisense-mediated axon skipping or genome editing, aiming to restore protein function in a mutation-specific manner. Animal models can facilitate the development of personalized medicine approaches; however, given that they target human mutations and therefore human genetic sequences, scientists rely on the availability of humanized animal models. Here, we outline the usefulness, caveats and potential of such models, using the example of the hDMDdel52/mdx model, a humanized model recently generated for Duchenne muscular dystrophy (DMD).
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available