Emerging Preclinical Animal Models for FSHD

Overview

Journal Trends Mol Med

Specialties General Medicine
Molecular Biology

Date 2015 Mar 25

PMID 25801126

Citations 30

Authors

Angela Lek

Fedik Rahimov

Peter L Jones

Louis M Kunkel

Affiliations

Soon will be listed here.

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.

Citing Articles

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