Gaining insights from transcriptomic signatures in spinal muscular atrophy: identification and exploration of new therapeutic targets
<p>5q-spinal muscular atrophy (SMA) is a rare, autosomal recessive, progressive, and lethal neuromuscular disease, most frequently of paediatric onset, caused by the loss of function of the survival of motor neuron (<i>SMN1</i>) gene. <i>SMN1</i> codes for the SMN prote...
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| Formato: | Thesis |
| Idioma: | English |
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2022
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