Testing kidney organoids for genome editing safety and effectiveness
Vascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editing
['FUNDING_U01'] · MASSACHUSETTS GENERAL HOSPITAL · NIH-10479164
This study is looking at tiny kidney models made from human cells to see how safe and effective gene editing tools, like CRISPR, can be used to fix genetic problems without causing harm, especially for people with conditions like Duchenne Muscular Dystrophy.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MASSACHUSETTS GENERAL HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-10479164 on ClinicalTrials.gov |
What this research studies
This research investigates the use of kidney organoids, which are miniaturized versions of kidneys created from human stem cells, to evaluate the safety and effectiveness of genome editing techniques like CRISPR/Cas9. By simulating human kidney tissue, the researchers aim to understand how these editing techniques can correct genetic mutations while minimizing harmful side effects. The study will focus on the delivery of genome editing tools via adeno-associated viruses and assess their impact on kidney cells, particularly in relation to conditions like Duchenne Muscular Dystrophy.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders, particularly those related to kidney function or Duchenne Muscular Dystrophy.
Not a fit: Patients with conditions unrelated to genetic mutations or those not involving kidney function may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective treatments for genetic diseases by ensuring that genome editing techniques do not cause unintended harm to patients.
How similar studies have performed: Other research has shown promise in using organoids for drug testing and genetic research, indicating that this approach may yield valuable insights.
Where this research is happening
BOSTON, UNITED STATES
- MASSACHUSETTS GENERAL HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MORIZANE, RYUJI — MASSACHUSETTS GENERAL HOSPITAL
- Study coordinator: MORIZANE, RYUJI
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.