| 摘要 |
The disclosure provides compounds, compositions, and methods for promoting motor neuron survival, and for treating or preventing neurodegenerative disorders, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). In accordance with an embodiment of the present invention, a method of regulating survival of motor neuron (SMN) protein levels in a cell is provided. The method includes contacting the cell with an effective amount of an agent which inhibits the level or activity of Nedd8 activating enzyme (NAE) in the cell and an effective amount of an agent which increases the level of SM7N protein, thereby regulating SMN protein levels in the cell. |
