Editing genes to treat metabolic diseases before and after birth
Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
This study is exploring new ways to use gene editing to help people with metabolic diseases, both before they are born and after, by fixing harmful genes with a special technology called CRISPR.
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 ID | NIH-10896255 on NIH RePORTER |
What this research studies
This research focuses on developing innovative gene editing techniques to treat metabolic diseases both before and after birth. Using advanced CRISPR technology, the team aims to deliver precise genetic modifications to patients, potentially correcting harmful mutations that lead to these diseases. The approach includes both in vivo editing, which targets cells within the body, and in utero editing, which could prevent genetic disorders from manifesting in newborns. By utilizing adeno-associated virus (AAV) vectors for delivery, the research seeks to overcome previous challenges in effectively administering gene therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with known genetic metabolic disorders, as well as pregnant women whose fetuses may be at risk for such conditions.
Not a fit: Patients with metabolic diseases that are not caused by genetic mutations or those who are not pregnant may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that prevent or significantly reduce the impact of metabolic diseases in affected individuals.
How similar studies have performed: While gene editing has shown promise in treating certain conditions, this specific approach of in utero editing for metabolic diseases is still largely untested and represents a novel frontier in the field.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Peranteau, William H. — Children's Hosp of Philadelphia
- Study coordinator: Peranteau, William H.
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.