Genetic circuitry of Survival motor neuron, the gene underlying spinal muscular atrophy.
Proc Natl Acad Sci U S A
; 110(26): E2371-80, 2013 Jun 25.
Article
em En
| MEDLINE
| ID: mdl-23757500
ABSTRACT
The clinical severity of the neurodegenerative disorder spinal muscular atrophy (SMA) is dependent on the levels of functional Survival Motor Neuron (SMN) protein. Consequently, current strategies for developing treatments for SMA generally focus on augmenting SMN levels. To identify additional potential therapeutic avenues and achieve a greater understanding of SMN, we applied in vivo, in vitro, and in silico approaches to identify genetic and biochemical interactors of the Drosophila SMN homolog. We identified more than 300 candidate genes that alter an Smn-dependent phenotype in vivo. Integrating the results from our genetic screens, large-scale protein interaction studies, and bioinformatic analysis, we define a unique interactome for SMN that provides a knowledge base for a better understanding of SMA.
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Base de dados:
MEDLINE
Assunto principal:
Proteínas de Ligação a RNA
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Genes de Insetos
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Proteínas de Drosophila
Limite:
Animals
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Humans
Idioma:
En
Ano de publicação:
2013
Tipo de documento:
Article