Nerve-cell defense mechanism may hold key to SBMA treatment
A defense mechanism called “autophagy” that neurons (nerve cells) use to protect themselves from dangerous misfolded proteins may hold the key to developing treatments for spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) and perhaps similar neurodegenerative diseases, new research shows.
Can toxic genes be blocked to treat disease?
Since the 1990s, gene therapy - the insertion of functional genes to compensate for nonfunctional ones - has been the goal of researchers working in several muscular dystrophies, spinal muscular atrophy, Friedreich's ataxia, metabolic muscle diseases and myotubular myopathy.
Stem cells restore movement in rats; may apply in SMA, ALS
A research team led by MDA grantee Douglas Kerr at Johns Hopkins University in Baltimore has developed a multistep regimen that improves the functional effect of transplanted stem cells in paralyzed rats and could have implications for motor neuron diseases like spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).