The genetic disorder Fragile X syndrome occurs when individuals don't make the Fragile X protein known as FMRP. Essential for normal brain development, FMRP helps control when and where proteins are ...

Researchers at Cincinnati Children's have demonstrated that AAV-mediated delivery of the human FMR1 gene can restore FMRP ...

Numerous potential treatments for neurological conditions, including autism spectrum disorders, have worked well in lab mice but then disappointed in humans. What would help is a noninvasive, ...

Salk Institute scientists find correcting dysregulations in star-shaped brain cells called astrocytes improves some fragile X syndrome symptoms, making the cells potential future therapeutic targets ...

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant ...

UCLA Health researchers have identified a potential drug target for treating Fragile X syndrome, the most common genetic cause of intellectual disability and autism that affects roughly 1 in 2,000 ...

UC Davis MIND Institute researchers Randi and Paul Hagerman are calling for increased awareness and screening for fragile X-associated conditions. In a new paper published in the New England Journal ...