The safety of gene therapy for Fabry disease has been proven

A safety study was conducted by using genetically engineered hematopoietic stem cells with a lentiviral vector that deliver a normal copy of the GLA gene. There were no adverse events associated with the use of the experimental drug, but key biochemical markers showed significant improvement. As a result, 3 out of 5 patients completely abandoned standard enzyme therapy.

Fabry disease is a disease of the group of lysosomal storage diseases and it is caused by a mutation in the GLA gene. This leads to severe metabolic disorders resulting to the accumulation of complex molecules in the body. The primary pathogenetic link in Fabry disease is a deficiency of α-galactosidase A, which normally cleaves the terminal residue of α-galactose of the oligosaccharide chain of neutral glycosphingolipids.

Currently, only substitution therapy is available to patients with this disease. This helps to reduce the level of toxic compounds, but such therapy does not eliminate metabolic disorders, so the disease is still progressing.

Source: MEDICAL XPRESS