Rett Syndrome: A History of Research and Therapeutic Outlooks
Type
Blog Post
...individuals with Rett syndrome experience regression of previously acquired skills such as speech, coordination,...landmark paper in Science showed that dramatic reversal of disease symptoms in mice is possible (Guy et... halted the neurological damage but completely reversed it, regardless of age or symptom severity. This... exciting finding injected hope that a similar reversal may be possible for children and adults with Rett...RNA trans-splicing, and protein replacement to reverse the impacts of this disease.
As a non-degenerative...non-degenerative, monogenic disease that has shown reversibility of symptoms in animal models, Rett syndrome is ...Guy J, Gan J, Selfridge J, Cobb S, Bird A (2007) Reversal of Neurological Defects in a Mouse Model of Rett...