Modeling the phenotype of spinal muscular atrophy by the direct conversion of human fibroblasts to motor neurons

Abstract

Spinal muscular atrophy (SMA) is a lethal autosomal recessive neurological disease characterized by selective degeneration of motor neurons in the spinal cord. In recent years, the development of cellular reprogramming technology has provided an alternative and effective method for obtaining patient-specific neurons in vitro. In the present study, we… (More)
DOI: 10.18632/oncotarget.14641

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