… splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy[…
Treatment of spinal muscular atrophy by sodium butyrate[J]. Proc Natl Acad Sci USA, 2001, …

… the spinal cord and cerebellum, muscle atrophy, motor impairment, neuromuscular junction
… and genetic modifiers of pathogenic mutations, as well as developing model therapies. The …

… We were able to rule out welldescribed genetic modifiers, such as EPHA4 and UNC13A, …
protein VAPB causes late-onset spinal muscular atrophy and amyotrophic lateral sclerosis. Am …

… survival motor neuron protein- dependent modifier for the rescue of spinal muscular atrophy
… dependent modifier for the rescue of spinal muscular atrophy Spinal muscular atrophy (SMA…

… genetic modifiers required for efficient expression of expanded CTG repeats using genome-wide
RNAi approach. 13 genes … Our results showed that RNAi treatment of F11C3.1 or RFC-…

… in spinal muscular atrophy (SMA), … genetic modifiers may also be involved in NEDCAM
manifestation. In particular, the effects of genetic modifiers are of unique interest given their effect …

… After the insurgence that is characterized by typical symptoms such as weakness in the limbs
and muscle twitches, the disease rapidly evolves into progressive muscle atrophy, paralysis…

… Genetic screens conducted in flies have shown Hippo as a genetic modifier of … model
reported MST1 as a key player in enhancing fast skeletal muscle atrophy. It reports …

… hereditary neuromuscular disease, spinal muscular atrophy (… survival motor neuron 1 (SMN1)
gene. It leads to a decrease … muscle atrophy resulting in the progressive muscle weakness, …

… muscular atrophy manifests mainly as a lower motor neuron disease. Why this is so and
whether other cell types contribute to the classic spinal muscular atrophy … the modifiers in SMA …