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Modeling the differential phenotypes of spinal muscular atrophy with high-yield generation of motor neurons from human induced pluripotent stem cells

Spinal muscular atrophy (SMA) is a devastating motor neuron disease caused by mutations of the survival motor neuron 1 (SMN1) gene. SMN2, a paralogous gene to SMN1, can partially compensate for the loss of SMN1. On the basis of age at onset, highest motor function and SMN2 copy numbers, childhood-on...

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Detalles Bibliográficos
Autores principales: Lin, Xiang, Li, Jin-Jing, Qian, Wen-Jing, Zhang, Qi-Jie, Wang, Zhong-Feng, Lu, Ying-Qian, Dong, En-Lin, He, Jin, Wang, Ning, Ma, Li-Xiang, Chen, Wan-Jin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Impact Journals LLC 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5522047/
https://www.ncbi.nlm.nih.gov/pubmed/28159932
http://dx.doi.org/10.18632/oncotarget.14925