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Fragile X Syndrome

How a dynein subunit maintains neuronal health

9 November 2023 | By Ellen Capon (Drug Target Review)

Understanding how a molecular transport system inside neurons works could be used for therapeutic approaches to neurodegenerative disease.

news

Novel approach stimulates DNA repair mechanisms to combat a leading cause of autism spectrum disorders

22 May 2023 | By Izzy Wood (Drug Target Review)

US researchers report promising results for fragile X syndrome, by stimulating cells' DNA repair mechanisms that could correct the inherited genetic defect associated with the disorder.

hand holding a section of a DNA strand above the remaining strand with forceps - idea of gene editing

news

TALEN is a more efficient gene editing tool for tightly packed DNA, finds study

28 January 2021 | By Hannah Balfour (Drug Target Review)

TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.

news

Mavoglurant could improve eye gaze in fragile X patients

25 January 2019 | By Iqra Farooq (Drug Target Review)

A first stage trial involving the experimental drug mavoglurant has shown early success in the treatment of gaze avoidance in fragile X syndrome patients...

news

Class of neurological disorders share 3D genome folding pattern

19 September 2018 | By Drug Target Review

Researchers have found another common thread linking nearly all of the TNR expansion diseases...

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Recommended

Fragile X Syndrome

How a dynein subunit maintains neuronal health

Novel approach stimulates DNA repair mechanisms to combat a leading cause of autism spectrum disorders

TALEN is a more efficient gene editing tool for tightly packed DNA, finds study

Mavoglurant could improve eye gaze in fragile X patients

Class of neurological disorders share 3D genome folding pattern