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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Publicat a:Proc Natl Acad Sci U S A
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2017
Matèries:
PNAS Plus
Accés en línia: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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