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Novel protective modifiers in mouse models of spinal muscular atrophy
(University of Missouri--Columbia, 2018)
[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Spinal Muscular Atrophy (SMA) is a neuromuscular disease caused by the homozygous deletion or mutation in the survival motor neuron-1 (SMN1) gene ...
Contribution of the immune system and astrocytes to spinal muscular atrophy pathology
(University of Missouri--Columbia, 2018)
[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease and the leading genetic cause of infant mortality. SMA is caused by ...
Novel mouse models for spinal muscular atrophy with respiratory distress type I : characterizing disease relevant models and evaluating therapeutic potential
(University of Missouri--Columbia, 2021)
Spinal muscular atrophy with respiratory distress type I (SMARD1) is a rare neurodegenerative disease caused by loss-of-function mutations in the immunoglobulin µ-DNA binding protein 2 (IGHMBP2). SMARD1 is defined by severe ...