Tay-Sachs disease

Bernard Sachs (1858-1944)

Bernard Sachs (1858-1944)

Bernard Sachs studied nervous system disorders in children in the United States during the nineteenth and twentieth centuries. In the late 1880s, Sachs described the fatal genetic neurological disorder called amaurotic family idiocy, later renamed Tay-Sachs disease. The disorder degrades motor skills as well as mental abilities in affected individuals. The expected lifespan of a child with

Gene Transfer Strategy Used to Treat Tay - Sachs Disease (2005), by Sabata Martino’s Research Group

A Direct Gene Transfer Strategy via Brain Internal Capsule Reverses the Biochemical Defect in Tay-Sachs Diseases (2005)

In the early 2000s, Sabata Martino and a team of researchers in Italy and Germany showed that they could reduce the symptoms of Tay-Sachs in afflicted mice by injecting them with a virus that infected their cells with a gene they lacked. Tay-Sachs disease is a fatal degenerative disorder that occurs in infants and causes rapid motor and mental impairment, leading to death at

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