Investigating new treatments for Mucopolysaccharidosis Type 1 in children.
Official title: PROJECT 3: MUCOPOLYSACCHARIDOSIS TYPE 1 (MPS1)
Funded at Children's Hosp of Philadelphia · NIH-10896264 · Data updated 2026-11-12
This study is looking at new ways to treat Mucopolysaccharidosis Type 1 (MPSI), a genetic disorder in children, by using advanced techniques that might fix the genetic problem, and if successful, there could be chances for patients to join clinical trials for these new treatments.
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) | $688,528 |
| NIH project number | 5U19NS132301-02 |
| Project ID | NIH-10896264 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for participation are children diagnosed with Mucopolysaccharidosis Type 1, particularly those with the W402X mutation.
✕Less likely to help
Patients with other types of Mucopolysaccharidosis or those without the specific genetic mutation targeted by this research may not benefit.
What this research studies
This research focuses on Mucopolysaccharidosis Type 1 (MPSI), a serious genetic disorder that affects children and can lead to severe health complications if untreated. The project aims to develop innovative treatments using adenine base editing techniques, which could potentially correct the genetic mutation responsible for the disease. The research will explore both postnatal and prenatal treatment options, with plans to initiate clinical trials if initial results are promising. Patients may have the opportunity to participate in these trials, contributing to the advancement of effective therapies.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for children with MPSI, potentially improving their quality of life and survival rates.
How similar studies have gone: Previous research has shown promise in using gene editing techniques for similar genetic disorders, indicating potential for success in this novel approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Davidson, Beverly L. — Children's Hosp of Philadelphia
- Study coordinator: Davidson, Beverly L.
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.