Analyzing unintended effects of gene editing therapies
Official title: OFF-TARGET RESOURCE CORE
Funded at Children's Hosp of Philadelphia · NIH-10896257 · Data updated 2026-11-12
This study is looking at how gene editing treatments for genetic disorders, like phenylketonuria and others, might affect your genes in ways we didn't expect, to help make sure these therapies are safe and effective for everyone.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $719,196 |
| NIH project number | 5U19NS132301-02 |
| Project ID | NIH-10896257 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with genetic disorders such as phenylketonuria, hereditary tyrosinemia type 1, or mucopolysaccharidosis type I.
✕Less likely to help
Patients with conditions unrelated to the genetic disorders being studied may not benefit from this research.
What this research studies
This research focuses on understanding the unintended effects of gene editing therapies, which are crucial for developing safe and effective treatments for genetic disorders like phenylketonuria, hereditary tyrosinemia type 1, and mucopolysaccharidosis type I. The project will assess both on-target and off-target editing activities, including their frequency and location, while also evaluating the overall genomic integrity and biological consequences of these edits. By providing essential analyses and services, this research aims to support the development of new gene therapies that meet regulatory standards.
Why it matters
Potential benefit: If successful, this research could lead to safer gene therapies with fewer unintended effects, improving treatment outcomes for patients with genetic disorders.
How similar studies have gone: Previous research has shown promise in understanding off-target effects in gene editing, indicating that this approach is both relevant and necessary for advancing gene therapy.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Wang, Xiao — Children's Hosp of Philadelphia
- Study coordinator: Wang, Xiao
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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.