Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. [electronic resource]
- Human molecular genetics Mar 2016
- 964-75 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1460-2083
10.1093/hmg/ddv629 doi
Adolescent Adult Age of Onset Animals Dependovirus--genetics Disease Models, Animal Follistatin--pharmacology Gene Deletion Genetic Vectors--chemistry Humans Immunologic Factors--pharmacology Mice Motor Neurons--drug effects Muscular Atrophy, Spinal--drug therapy Myostatin--antagonists & inhibitors Phenotype RNA Splicing--drug effects Survival of Motor Neuron 1 Protein--genetics Survival of Motor Neuron 2 Protein--agonists