Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by progressive motor neuron loss and caused by mutations in SMN1 (Survival Motor Neuron 1). The disease severity inversely correlates with the copy number of SMN2, a duplicated gene that is nearly identical to SMN1. We have d...

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Detalhes bibliográficos
Publicado no:Proc Natl Acad Sci U S A
Main Authors: Woo, Caroline J., Maier, Verena K., Davey, Roshni, Brennan, James, Li, Guangde, Brothers, John, Schwartz, Brian, Gordo, Susana, Kasper, Anne, Okamoto, Trevor R., Johansson, Hans E., Mandefro, Berhan, Sareen, Dhruv, Bialek, Peter, Chau, B. Nelson, Bhat, Balkrishen, Bullough, David, Barsoum, James
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2017
Assuntos:
PNAS Plus
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5338378/
https://ncbi.nlm.nih.gov/pubmed/28193854
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1616521114
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