Premium
Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition
Author(s) -
Narver Heather L.,
Kong Lingling,
Burnett Barrington G.,
Choe Dong W.,
BoschMarcé Marta,
Taye Addis A.,
Eckhaus Michael A.,
Sumner Charlotte J.
Publication year - 2008
Publication title -
annals of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.764
H-Index - 296
eISSN - 1531-8249
pISSN - 0364-5134
DOI - 10.1002/ana.21449
Subject(s) - trichostatin a , spinal muscular atrophy , histone deacetylase inhibitor , atrophy , medicine , histone deacetylase , muscle atrophy , progressive muscular atrophy , endocrinology , pathology , disease , biology , histone , amyotrophic lateral sclerosis , gene , biochemistry
Early treatment with the histone deacetylase inhibitor, trichostatin A, plus nutritional support extended median survival of spinal muscular atrophy mice by 170%. Treated mice continued to gain weight, maintained stable motor function, and retained intact neuromuscular junctions long after trichostatin A was discontinued. In many cases, ultimate decline of mice appeared to result from vascular necrosis, raising the possibility that vascular dysfunction is part of the clinical spectrum of severe spinal muscular atrophy. Early spinal muscular atrophy disease detection and treatment initiation combined with aggressive ancillary care may be integral to the optimization of histone deacetylase inhibitor treatment in human patients. Ann Neurol 2008; 64:465–470