IBCCES is the global leader in training and certification for healthcare professionals, educators and corporate partners who work with individuals with autism and other cognitive disorders. Our reach extends to more than 2 million people in all 50 states and over 70 countries around the globe. IBCCES Member Learning Community is provided as a free service to all IBCCES members who have completed one or more of our training and certification programs.
Antisense therapy restores fragile X protein production in human cells
An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented in the study, that aberrant alternative splicing of messenger RNA (mRNA) plays a principal role in fragile X syndrome, the most common form of inherited intellectual disability and the most frequent single-gene cause of autism.