Understanding how long noncoding RNAs affect phenylketonuria
Official title: Molecular Mechanism of long Noncoding RNAs in Phenylketonuria
Funded at University of Tx Md Anderson Can Ctr · NIH-11015069 · Data updated 2026-11-12
This study is looking at how certain molecules in our genes, called lncRNAs, might help find new ways to treat phenylketonuria (PKU), a condition caused by changes in a specific gene, and it could lead to better options for people living with PKU.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Tx Md Anderson Can Ctr NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | April 10, 2023 |
| Project end | January 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $472,897 |
| NIH project number | 5R01HD110520-03 |
| Project ID | NIH-11015069 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with phenylketonuria, particularly those with specific genetic variants affecting the PAH gene.
✕Less likely to help
Patients with phenylketonuria who do not have the relevant genetic variants or those with other metabolic disorders may not benefit from this research.
What this research studies
This research investigates the role of long noncoding RNAs (lncRNAs) in the genetic disorder phenylketonuria (PKU), which is caused by mutations in the phenylalanine hydroxylase (PAH) gene. By studying the interactions between lncRNAs and enzymes, the research aims to uncover new therapeutic strategies for PKU. The team has already shown that specific lncRNAs can influence enzyme activity and reduce harmful levels of phenylalanine in a mouse model. This work could lead to innovative treatments for patients with PKU who currently have limited options.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that effectively lower phenylalanine levels in PKU patients, improving their health outcomes.
How similar studies have gone: Previous research has shown promise in using lncRNAs for therapeutic purposes in genetic diseases, indicating that this approach may be viable.
Where this research is happening
-
University of Tx Md Anderson Can Ctr
Houston, United States Active
Researchers
- Principal investigator: Yang, Liuqing — University of Tx Md Anderson Can Ctr
- Study coordinator: Yang, Liuqing
Related research
Other NIH-funded projects on the same topics.
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LNP-based adenine base editing for PKU and urea cycle disorders
A front-line, patient-focused research project aims to develop safe gene-editing therapies that could lessen or correct the biochemical problems in PKU and related urea cycle disorders.
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PKU: a new gene-editing treatment for a common PAH mutation
A new therapy using lipid nanoparticles to correct a common PKU mutation could reduce the need for strict diets and dangerous enzyme injections.
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A new gene-editing approach to treat phenylketonuria (PKU)
A medical research project is testing a gene-editing strategy using AAV-delivered CRISPR tools to correct PAH gene defects in PKU patients.
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Phenylalanine and brain function in PKU carriers and non-carriers
We’re exploring how a single elevated nutrient (phenylalanine) affects thinking and brain function in adults who carry a PKU gene variant compared with those who don’t.
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Gene-editing treatment for phenylketonuria caused by the P281L PAH mutation
A lipid nanoparticle-delivered adenine base editor to correct the P281L PAH mutation in people with phenylketonuria.
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Using gene editing to treat phenylketonuria
This study is exploring a new way to use gene editing to help people with phenylketonuria (PKU) by fixing the genetic issues that cause the condition, aiming to offer a better treatment than the current strict diets and medications.
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.