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Emerging preclinical animal models for FSHD.
- Source :
-
Trends in Molecular Medicine . May2015, Vol. 21 Issue 5, p295-306. 12p. - Publication Year :
- 2015
-
Abstract
- Facioscapulohumeral dystrophy (FSHD) is a unique and complex genetic disease that is not entirely solved. Recent advances in the field have led to a consensus genetic premise for the disorder, enabling researchers to now pursue the design of preclinical models. In this review we explore all available FSHD models (DUX4-dependent and -independent) for their utility in therapeutic discovery and potential to yield novel disease insights. Owing to the complex nature of FSHD, there is currently no single model that accurately recapitulates the genetic and pathophysiological spectrum of the disorder. Existing models emphasize only specific aspects of the disease, highlighting the need for more collaborative research and novel paradigms to advance the translational research space of FSHD. [ABSTRACT FROM AUTHOR]
Details
- Language :
- English
- ISSN :
- 14714914
- Volume :
- 21
- Issue :
- 5
- Database :
- Academic Search Index
- Journal :
- Trends in Molecular Medicine
- Publication Type :
- Academic Journal
- Accession number :
- 102318224
- Full Text :
- https://doi.org/10.1016/j.molmed.2015.02.011