Fragile X syndrome neurons restored using CRISPR/Cas9-guided activation strategy

(Whitehead Institute for Biomedical Research) Fragile X syndrome is the most frequent cause of intellectual disability in males, affecting 1 out of 3600 boys born. For the first time, researchers at Whitehead Institute have restored activity to the fragile X syndrome gene in affected neurons using a modified CRISPR/Cas9 system that removes the methylation--the molecular tags that keep the mutant gene shut off--suggesting that this method may be useful for targeting diseases caused by abnormal methylation.

from EurekAlert! - Social and Behavioral Science http://ift.tt/2GAMrAX