Research Reveals Underlying Pathology of Spinal Muscular Atrophy Disease (IMAGE)
Caption
Spinal muscular atrophy is the leading genetic causes of death for infants, but there is currently no approved treatment. A team of scientists from Cold Spring Harbor Laboratory has a potential drug to treat the disease that is injected into the cerebrospinal fluid of patients. But the researchers have discovered that in mouse models the drug is effective even when injected subcutaneously, and it is not necessary in the central nervous system. Shown here are spinal sections from three different mice with spinal muscular atrophy. Systemic drug treatment (middle panel) increases the presence of motor neurons (red spots) over the untreated mice (left panel). Surprisingly, the results are very similar when treatment is excluded from the central nervous system (right panel), suggesting a possible new path for spinal muscular atrophy drug treatment.
Credit
A. Krainer/ Cold Spring Harbor Laboratory
Usage Restrictions
None
License
Licensed content