Browsing Veterinary Pathobiology electronic theses and dissertations (MU) by Thesis Semester "2009 Spring"
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Molecular genetics of spinal muscular atrophy: insights into various routes of therapeutic intervention
(University of Missouri--Columbia, 2009)Spinal Muscular Atrophy is an autosomal recessive neuromuscular disorder that is the leading genetic cause of infant mortality. In humans, a nearly identical copy gene is present called SMN2 that is retained in all SMA ... -
Spinal muscular atrophy and the therapeutic application of trans-splicing RNA
(University of Missouri--Columbia, 2009)Development of RNA modalities to re-direct pathogenic pre-mRNA splicing events is the key to curing monogenic diseases. Improving the efficiency of these molecules in vivo is critical as they move towards clinical applications. ...