Gene-Based Therapy Restores Cellular Development and Function in Brain Cells From People With Timothy Syndrome

Not only do these findings offer a potential road map to treat Timothy syndrome, but research into this condition also offers broader insights into other rare genetic conditions and mental disorders (NIH Director).

Researchers demonstrated the effectiveness of using antisense oligonucleotides (ASOs) to restore cellular deficits underlying Timothy syndrome in human brain tissue structures and organoids (Study Lead).

Read at National Institute of Mental Health (NIMH)