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...
| Main Author: | |
|---|---|
| Other Authors: | |
| Format: | Thesis |
| Language: | English |
| Published: |
2022
|
| Subjects: |