sly syndrome การใช้

Birkenmeier and his colleagues reported in 1992 that gene therapy had corrected the defects caused by Sly syndrome in mice.
The mouse model has been used to test experimental therapies for Sly syndrome and for closely related disorders like Tay-Sachs disease and Gaucher's disease.
For instance, Birkenmeier developed a mouse model for a very rare inherited human disorder, Sly syndrome, that causes cardiac murmurs and mental retardation and often brings death in childhood.
However, the condition may be present in association with other syndromes : Turner syndrome, Noonan syndrome, Loeys-Dietz syndrome, Marfan syndrome, Ehlers-Danlos syndrome, Morquio syndrome, trisomy 18, trisomy 21, homocystinuria, osteogenesis imperfecta, multiple lentigines syndrome ( LEOPARD syndrome ), Sly syndrome ( mucopolysaccharidosis type VII ), and scoliosis.