Analyzing unintended effects of gene editing therapies
OFF-TARGET RESOURCE CORE
['FUNDING_OTHER'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-10896257
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-10896257 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with genetic disorders such as phenylketonuria, hereditary tyrosinemia type 1, or mucopolysaccharidosis type I.
Not a fit: Patients with conditions unrelated to the genetic disorders being studied may not benefit from this research.
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 performed: 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
PHILADELPHIA, UNITED STATES
- CHILDREN'S HOSP OF PHILADELPHIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WANG, XIAO — CHILDREN'S HOSP OF PHILADELPHIA
- Study coordinator: WANG, XIAO
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.