Novel myosin-based therapies for congenital cardiac and skeletal myopathies
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Novel myosin-based therapies for congenital cardiac and skeletal myopathies. / Ochala, Julien; Sun, Yin Biao.
In: Journal of Medical Genetics, Vol. 53, No. 10, 01.10.2016, p. 651-654.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Novel myosin-based therapies for congenital cardiac and skeletal myopathies
AU - Ochala, Julien
AU - Sun, Yin Biao
PY - 2016/10/1
Y1 - 2016/10/1
N2 - The dysfunction in a number of inherited cardiac and skeletal myopathies is primarily due to an altered ability of myofilaments to generate force and motion. Despite this crucial knowledge, there are, currently, no effective therapeutic interventions for these diseases. In this short review, we discuss recent findings giving strong evidence that genetically or pharmacologically modulating one of the myofilament proteins, myosin, could alleviate the muscle pathology. This should constitute a research and clinical priority.
AB - The dysfunction in a number of inherited cardiac and skeletal myopathies is primarily due to an altered ability of myofilaments to generate force and motion. Despite this crucial knowledge, there are, currently, no effective therapeutic interventions for these diseases. In this short review, we discuss recent findings giving strong evidence that genetically or pharmacologically modulating one of the myofilament proteins, myosin, could alleviate the muscle pathology. This should constitute a research and clinical priority.
KW - Cardiomyopathy
KW - Congenital heart disease
KW - Muscle disease
UR - http://www.scopus.com/inward/record.url?scp=84978871066&partnerID=8YFLogxK
U2 - 10.1136/jmedgenet-2016-103881
DO - 10.1136/jmedgenet-2016-103881
M3 - Journal article
C2 - 27412953
AN - SCOPUS:84978871066
VL - 53
SP - 651
EP - 654
JO - Journal of Medical Genetics
JF - Journal of Medical Genetics
SN - 0022-2593
IS - 10
ER -
ID: 245662454