Article
Identification of a battery of tests for drug candidate evaluation in the SMNDelta7 neonate model of spinal muscular atrophy.
Experimental neurology - 1 Jul 2008
El-Khodor Bassem F, Edgar Nicole, Chen Angela, Winberg Margaret L, Joyce Cynthia, Brunner Daniela, Suárez-Fariñas Mayte, Heyes Melvyn P
Abstract excerpt
Spinal muscular atrophy (SMA) is characterized by selective loss of alpha-motor neurons and is caused by homozygous loss or mutation in the survival motor neuron (SMN1) gene. Loss of SMN1 is partially compensated by the copy gene, SMN2. Currently, there are no specific treatments for SMA. Key features of SMA are modeled in mice by deletion of murine Smn, and insertion of both full length human SMN2 gene and the...
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