Gene editing in the brain using a new delivery method
Gene editing in the brain with CRISPR-PEG
['FUNDING_R01'] · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · NIH-11015029
This study is testing a new way to deliver gene editing tools to brain cells to help treat fragile X syndrome, a condition that affects learning and development, using a special delivery system that has shown good results in animal tests.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF TEXAS HLTH SCIENCE CENTER (nih funded) |
| Locations | 1 site (SAN ANTONIO, UNITED STATES) |
| Trial ID | NIH-11015029 on ClinicalTrials.gov |
What this research studies
This research investigates a novel gene editing delivery system called CRISPR-PEG, designed to effectively deliver CRISPR technology to brain cells. The approach focuses on treating fragile X syndrome, a genetic condition that leads to intellectual disabilities, by using a biocompatible vehicle that enhances the delivery of gene editing tools directly to neurons. The researchers have shown promising results in animal models, demonstrating the ability to edit neurons with high specificity after administration. This innovative method aims to overcome previous challenges associated with delivering gene therapies to the brain.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with fragile X syndrome, particularly those with the genetic markers associated with the condition.
Not a fit: Patients with brain disorders not related to fragile X syndrome or those without the specific genetic markers may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with fragile X syndrome, potentially improving cognitive function and quality of life.
How similar studies have performed: While gene editing has shown promise in various contexts, this specific approach using CRISPR-PEG for brain delivery in fragile X syndrome is novel and has not been extensively tested in humans.
Where this research is happening
SAN ANTONIO, UNITED STATES
- UNIVERSITY OF TEXAS HLTH SCIENCE CENTER — SAN ANTONIO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LEE, HYE YOUNG — UNIVERSITY OF TEXAS HLTH SCIENCE CENTER
- Study coordinator: LEE, HYE YOUNG
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.
Conditions: autism-fragile X (AFRAX) syndrome