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Survival Motor Neuron 2 pre-mRNA (None)

Target: None
Molecular classification: RNA, Precursor mRNA

Overview

Survival motor neuron 2 pre-mRNA (SMN2 pre-mRNA) is the precursor messenger RNA transcribed from the SMN2 gene. The SMN2 gene is a nearly identical copy of the SMN1 gene, both of which encode the survival motor neuron (SMN) protein. This protein is essential for cellular processes, particularly in motor neurons, and its deficiency leads to spinal muscular atrophy (SMA). Therapies aim to modulate alternative splicing at exon 7 to increase production of full-length, functional SMN protein.

Other names: SMN2 pre-mRNASMN2 precursor mRNA

Mechanism of action

Splice modulation to increase exon 7 inclusion

Biological functions

Pre-mRNA splicingsnRNP assemblyTemplate for SMN protein production

Disease associations

Spinal muscular atrophy

Interacting drugs

Risdiplam

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