18751096. THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER (University of Massachusetts)
Contents
THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
Organization Name
Inventor(s)
Joel D. Richter of Wellesley MA (US)
Sneha Shah of Worcester MA (US)
Jonathan Watts of Worcester MA (US)
THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
This abstract first appeared for US patent application 18751096 titled 'THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
Original Abstract Submitted
Provided herein, in various embodiments, are methods of treating a fragile X-associated disorder (e.g., fragile X syndrome), comprising administering to a subject in need thereof, a therapeutically effective amount of an agent that decreases expression of an aberrant fragile X messenger ribonucleoprotein 1 (FMR1) gene product (e.g., FMR1-217). Also provided herein, in various embodiments, are compositions (e.g., polynucleotides such as antisense oligonucleotides or pharmaceutical compositions) for decreasing expression of an aberrant FMR1 gene product.
