Mapping how autism-linked genes change brain cells and circuits

In vivo Perturb-map: scalable genetic screens with single-cell and spatial resolution in intact tissues

NIH-funded research Scripps Research Institute, the · NIH-11252335

Researchers are building tools to see which brain cell types and connections change when autism-linked genes are altered, to help guide future treatments for people with autism.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionScripps Research Institute, the NIH-funded
Lab location1 site (La Jolla, United States)
Project IDNIH-11252335 on NIH RePORTER

What this research studies

Scientists will create a method called Perturb-map that makes many small genetic changes in brains and then reads out which change happened in each cell while keeping the whole brain intact. They will use CRISPR to introduce edits, clear whole-brain tissue, and perform repeated light-sheet imaging plus molecular detection to find the location, cell type, shape, and projection patterns of each altered cell. By linking specific gene perturbations to cell identities and wiring patterns, the team aims to tell whether different autism risk genes affect the same brain regions or distinct circuits. This is preclinical lab work done in tissue models rather than a patient treatment trial, but it is intended to point to targets for future therapies.

Who could benefit from this research

Good fit: This project does not enroll patients; it is preclinical laboratory research using animal and tissue models and is not open to patient volunteers.

Not a fit: People seeking immediate new treatments or wishing to enroll in a clinical trial will not receive direct benefit from this laboratory-focused project in the near term.

Why it matters

Potential benefit: If successful, the work could identify the specific brain cells and circuits affected by autism genes, providing targets for future diagnostics or treatments.

How similar studies have performed: Related methods like single-cell CRISPR screens and spatial transcriptomics have shown promise, but combining pooled in vivo genetic perturbations with whole‑brain spatial readout is a novel and ambitious advance.

Where this research is happening

La Jolla, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Autistic Disorder
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.