GENETIC ENGINEERING: GENETIC DISEASES

The Fragile X Syndrome

Treat the symptoms of fragile X

How much can we modify the brain by behavioral therapy? Can the actual
shape and size of nerve cell connections be reversed by behavioral
intervention? Can behavioral symptoms be reversed by behavioral
intervention? Can symptoms be reversed by pharmacological
intervention? FRAXA awarded grants to Don Bailey, Elizabeth Dykens,
Mina Johnson-Glenberg, John Larson and Kenneth Mack to conduct studies
of the human symptoms of fragile X, and to Linda Crnic, Rich Paylor,
Frank Kooy, to pinpoint symptoms in the fragile X mouse model. If
therapies and educational strategies can be designed with the specific
needs of patients in mind, they are more likely to be effective.
Likewise, the more we understand specific symptoms and their causes in
fragile X syndrome, the more likely it is that drugs can be selected,
or discovered, that can target these symptoms. The new class of drugs
called Ampakines, is an example: FRAXA has funded a trial of Ampakines
in fragile X mice and now will fund a trial of this medication in
adults with fragile X, under the direction of Elizabeth Berry-Kravis.