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资源简介：
SMA patient with SMN1 Exon1 isolated deletion
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创建时间：
2023-10-22
相关数据集
Gene Expression Analysis of spinal cord obtained from a mouse model of severe Spinal Muscular Atrophy (SMA). Mus musculus
Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. No effective therapy is currently available.
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Binding to SMN2 pre-mRNA-Protein complex elicits specificity for small molecule splicing modifiers
Small molecule splicing modifiers have been extensively described which target the generic splicing machinery and thus have low target specificity. We have identified potent splicing modifiers with un
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Canine DUXC isoforms and functional similarity to human DUX4 [RNA-seq]
Human DUX4 and mouse Dux transcription factors are normally expressed in the germ line and early embryonic cells where they activate the cleavage stage genes. The misexpression of DUX4 in skeletal mus
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Gene Expression Analysis of laser-microdissected motorneurons in Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. Stem cell transplantation could represent a t
NIAID Data Ecosystem30
