Researchers University of Pennsylvania report the first ever use of gene therapy, in dogs, to cure a gene involving multiple body organs. Mucopolysaccharidosis VII (MPS) is one of a group of rare genetic disorders called lysosomal storage diseases. Other examples include Tay Sachs disease and Gaucher disease. MPS involves an enzyme defect, leading to blindness, heart disease, and loss of mobility.
Most humans with MPS die in childhood. Researchers at the University of Pennsylvania have carried out the first ever gene therapy for MPS in dogs born with the disease. This introduces a healthy version of the defective enzyme, beta-glucuronidase, into the animals' liver. Treated dogs gained weight normally, compared to untreated animals, and showed none of the expected health problems.
This is the first time gene therapy has been used for a disease involving multiple organs. It could replace the standard treatment - which is to give injections of the missing enzyme. The therapy could also be used to treat other lysosomal storage diseases.