Scientists found a way to restore brain cells
Imagine being able to fix a genetic glitch in the brain. That's exactly what scientists have done with a new drug for Timothy syndrome, a rare disorder that affects brain development. According to a study in Nature, a drug called antisense oligonucleotide enables human neurons to develop correctly even when they have the mutation associated with the disorder.
Timothy syndrome stems from a single gene mutation in a person's DNA. The new drug works by using an antisense nucleotide, a small synthetic piece of genetic material that changes the proteins produced by cells. Specifically, it replaces a faulty protein with a healthy one, effectively counteracting the mutation behind the disorder.
This method could potentially be applied to treat other genetic conditions, including some forms of schizophrenia, epilepsy, ADHD, and autism spectrum disorder.
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