Understanding the genetic factors involved in autism through neuron analysis
Unraveling the Genetic Programs Engaged in ASD Neurons Through Coupled Transcriptomic and Phenotypic Readouts
This study is looking at how genes might affect autism by examining brain cells made from stem cells, using special tools to change certain genes and see how it impacts the cells, which could help us understand autism better and improve treatments in the future.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-10876960 on NIH RePORTER |
What this research studies
This research investigates the genetic mechanisms underlying autism spectrum disorders (ASD) by studying human neurons derived from pluripotent stem cells. The team will use advanced techniques, including CRISPR technology, to manipulate genes associated with ASD and analyze how these changes affect neuron structure and function. By comparing a diverse set of ASD neuron samples with matched controls, the research aims to identify genetic signatures that correlate with specific neuronal characteristics. This comprehensive approach could lead to a better understanding of ASD and inform future clinical interventions.
Who could benefit from this research
Good fit: Ideal candidates for this research are children aged 0-11 years diagnosed with autism spectrum disorders.
Not a fit: Patients with autism who do not have identifiable genetic variants or those outside the specified age range may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new insights into the biological basis of autism, potentially guiding the development of targeted therapies.
How similar studies have performed: Previous research using similar genetic and cellular approaches has shown promise in understanding complex neurodevelopmental disorders, indicating potential for success in this area.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Farhi, Samouil — Broad Institute, INC.
- Study coordinator: Farhi, Samouil
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.