Neurons affected by the Fragile X syndrome can be rescued by CRISPR manipulations
Neurons affected by the Fragile X syndrome can be rescued by CRISPR manipulations - science news

Fragile X syndrome is a disease affecting one in 3,600 men, characterized by autistic-like symptoms and intellectual disabilities. The syndrome is caused by a mutation in a gene called FMR1. A new study reports that it is possible to restore this gene by using a genetic editing system based on CRISPR/Cas9. After correcting the mutation of the FMR1 gene, neurons in cell cultures returned to normal functioning. When these neurons were implanted in animals, the FMR1 gene remained active in the neurons for at least three months. The study shows that the disorder is reversible at neuronal level. However, further research is needed to demonstrate the efficacy of this approach in animals affected by Fragile X and ultimately in human patients.

Read the full story: MIT
Scientific publication: Cell