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

from National Institute of Mental Health (NIMH)1 month ago
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).
National Institute of Mental Health (NIMH)https://www.nimh.nih.gov/news/science-news/2024/gene-based-therapy-restores-cellular-development-and-function-in-brain-cells-from-people-with-timothy-syndrome
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).
National Institute of Mental Health (NIMH)https://www.nimh.nih.gov/news/science-news/2024/gene-based-therapy-restores-cellular-development-and-function-in-brain-cells-from-people-with-timothy-syndrome
Read at National Institute of Mental Health (NIMH)
#timothy-syndrome #genetic-disorder #therapy #organoids #nih
[
add
]
[
|
|
]