Understanding how Nemo-like Kinase affects blood cell development in Diamond Blackfan Anemia.
Characterization of the Role of Nemo-like Kinase in Normal and Diamond Blackfan Anemia Models of Erythropoiesis.
This study is looking at how a protein called NLK affects blood stem cells in people with Diamond Blackfan Anemia, to see if changing it can help these cells grow into healthy blood cells and improve treatments for patients like you.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-10895407 on NIH RePORTER |
What this research studies
This research investigates the role of Nemo-like Kinase (NLK) in Diamond Blackfan Anemia (DBA), a condition that leads to bone marrow failure and anemia. By using advanced techniques like CRISPR/Cas9, the study aims to knock out NLK in blood stem cells from DBA patients to see how this affects their ability to develop into healthy blood cells. The research will also explore the mechanisms behind NLK activation and its impact on blood cell production, potentially leading to new treatment strategies. Patients may benefit from insights that could improve stem cell transplantation outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Diamond Blackfan Anemia, particularly those with mutations affecting ribosomal genes.
Not a fit: Patients with forms of anemia unrelated to ribosomal gene mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new gene therapy approaches that improve treatment outcomes for patients with Diamond Blackfan Anemia.
How similar studies have performed: Previous research has shown promise in using CRISPR technology for gene therapy in similar conditions, indicating potential for success in this approach.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Wilkes, Mark Christopher — Stanford University
- Study coordinator: Wilkes, Mark Christopher
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.