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A high-throughput genome-wide RNAi screen identifies modifiers of survival motor neuron protein

1 May 2021

Abstract excerpt

Spinal muscular atrophy (SMA) is a debilitating neurological disorder marked by degeneration of spinal motor neurons and muscle atrophy. SMA results from mutations in survival motor neuron 1 (SMN1), leading to deficiency of survival motor neuron (SMN) protein. Current therapies increase SMN protein and improve patient survival but have variable improvements in motor function, making it necessary to identify...

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PMID: 33979606
DOI: 10.1016/j.celrep.2021.109125

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A high-throughput genome-wide RNAi screen identifies modifiers of survival motor neuron proteinPMID 33979606Read ↗

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