Cellular Transplantation

An alternative method of replacement is that of organ or
cellular transplantation, which aims at providing a permanent
functioning source of the missing gene product. This approach
has been applied to some inborn errors of metabolism, such as
mucopolysaccharidoses, using bone marrow transplantation
from matched donors. Again, the blood–brain barrier prevents
effective treatment of CNS manifestations of disease. The
potential for direct replacement of missing intracellular
enzymes in treating inborn errors of metabolism is also being
determined experimentally.