Exploring Genetic Factors in Autism Brain Cells
Unraveling the Genetic Programs Engaged in ASD Neurons Through Coupled Transcriptomic and Phenotypic Readouts
This project looks at genetic differences in brain cells from people with autism to understand how these differences affect brain cell function.
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-11129919 on NIH RePORTER |
What this research studies
Autism spectrum disorders (ASD) have many different genetic causes, and understanding how these genetic changes affect brain cells is a big challenge. This project uses special brain cells grown in the lab from human stem cells, which carry the unique genetic background of individuals. Researchers will use a precise genetic tool called CRISPR to turn off specific genes in these lab-grown brain cells to see how it changes their shape and electrical signals. They will also compare brain cells from a large group of people with and without ASD to find common genetic patterns. This work aims to connect specific genetic changes to how brain cells function in autism.
Who could benefit from this research
Good fit: This project uses donated human stem cells, so individuals who have contributed or may contribute biological samples for research on autism could be considered.
Not a fit: Patients will not directly receive treatment or intervention from this foundational laboratory research.
Why it matters
Potential benefit: If successful, this work could help identify new targets for future treatments by revealing the fundamental genetic mechanisms underlying autism.
How similar studies have performed: While the specific combination of advanced techniques is novel, using human stem cell-derived neurons to study autism genetics is a growing area of research.
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.