A combinatorial approach increases SMN level in SMA model mice.
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DOI: 10.1093/hmg/ddac068
Abstract: Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by reduced expression of the survival motor neuron (SMN) protein. Current disease-modifying therapies increase SMN levels and dramatically improve survival and motor function of SMA patients.… read more here.
Keywords: model mice; sma; increases smn;