Identifying epigenetic regulators of survival motor neuron protein to treat spinal muscular atrophy
<p>Spinal muscular atrophy (SMA) is a rare monogenic disease of mostly paediatric onset, affecting approximately 1:8,000 – 10,000 live births. If untreated, it leads to progressive muscle weakness and paralysis, due to degeneration of lower α-motor neurons in the spinal cord and brainstem. Thr...
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| Format: | Thesis |
| Language: | English |
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2022
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