6 Questions
What is the name of the genetic disorder that can be treated with the new drug?
Timothy syndrome
What is the purpose of the antisense nucleotide?
To replace a defective protein with a healthy version
What is the name of the professor who commented on the study?
Dr. Huda Zoghbi
What type of material is used to create the antisense nucleotide?
Synthetic genetic material
What is the potential application of this approach beyond Timothy syndrome?
For treating other genetic disorders, including schizophrenia, epilepsy, ADHD and autism spectrum disorder
What is the name of the journal where the study was published?
Nature
Study Notes
Restoring Brain Cells
- Scientists have discovered a way to repair brain cells impaired by a rare genetic disorder called Timothy syndrome.
- A drug called antisense oligonucleotide has been found to allow human neurons to develop normally despite carrying a mutation.
- Timothy syndrome is caused by a mutation of a single gene in a person's DNA.
- The antisense oligonucleotide is a small piece of synthetic genetic material that alters the proteins made by a cell.
- The drug works by replacing a defective protein with a healthy version, counteracting the mutation responsible for the disorder.
- This approach could potentially be used to treat other genetic disorders, including:
- Schizophrenia
- Epilepsy
- ADHD
- Autism spectrum disorder
Scientists have discovered a way to repair brain cells impaired by a rare genetic disorder called Timothy syndrome using a drug called antisense oligonucleotide.
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