Stephanie87-123 t1_j8rhbfd wrote
Reply to comment by TrenchantPergola in Recently a gene therapy for a rare inherited neurodegenerative disorder was approved. How come cure is achieved through bone marrow transplantation? by nickoskal024
The disease is not caused by ARSA deficiency in leukocytes specifically though, but rather by ARSA deficiency in all cells that lead to sulfatide accumulation that is more toxic for specific organs/cell types, like the brain and the galbladder. The bone marrow transplantations works because the blood derived cells will migrate into the brain and breakdown the excessive sulfatides in the extracellular space.
Viewing a single comment thread. View all comments