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Emerging preclinical animal models for FSHD.

Authors :
Lek, Angela
Rahimov, Fedik
Jones, Peter L.
Kunkel, Louis M.
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