We have several research stage ZFN-mediated gene-modification programs in progress. These initiatives include programs in monogenic diseases including hemoglobinopathies such as sickle cell anemia and immune system disorders such as X-linked severe combined immunodeficiency (X-linked SCID).
Mutations in the gene encoding the globin gene cause a variety of inherited conditions of the blood such as sickle cell disease and beta-thalassemia. Sangamo scientists have used ZFN-mediated gene correction in primary cells as an approach to correct such genetic lesions. With our collaborators we are developing these ZFNs for use in hematopoietic stem cells (HSCs) as potential therapeutics.
Lysosomal Storage Disorders
Lysosomal storage disorders are rare inherited metabolic disorders caused by malfunction of the lysosome, an organelle in cells that is required for breakdown and turnover of proteins and lipids. This dysfunction is usually a consequence of a single mutation in genes encoding specific lysosomal enzymes required for the metabolism of lipids, glycoproteins (sugar containing proteins) or mucopolysaccharides. Lysosomal storage diseases include disorders such as Gaucher disease, Fabry disease and Pompe disease.