Browsing Theses and Dissertations (MU) by Title "using SMN gene replacement to address biological questions"
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Gene therapy in mouse models of spinal muscular atrophy :
(University of Missouri--Columbia, 2013)Spinal Muscular Atrophy (SMA), an autosomal recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the functional, homozygous loss of the Survival Motor Neuron-1 (SMN1) gene ...