NIH-funded research NIH-funded research

Creating new CRISPR tools for gene editing therapy

Official title: Developing novel Type 1 CRISPR tools for gene editing therapy

Funded at Genetobe INC. · NIH-10821108 · Data updated 2026-11-12

This study is working on new CRISPR tools to make gene editing safer and more precise for people with sickle cell disease, aiming to create better treatments with fewer risks.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionGenetobe INC.
NIH-funded
Lab location1 site (Ann Arbor, United States)
Project startFebruary 15, 2024
Project endJanuary 31, 2026
Funding year2024
Award (this year)$293,319
NIH project number1R41GM153041-01
Project IDNIH-10821108 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates for this research are individuals with genetic disorders, particularly those affected by sickle cell disease.

Less likely to help

Patients with conditions not related to genetic disorders or those who do not have access to gene editing therapies may not benefit from this research.

What this research studies

This research focuses on developing innovative CRISPR tools to enhance gene editing therapies, particularly for conditions like sickle cell disease. By exploring Type I CRISPR systems, the project aims to improve the precision and safety of gene editing, addressing limitations of current methods that rely on Cas9. The approach involves using non-double-strand break techniques to minimize potential damage to DNA, which could lead to safer treatments for patients. If successful, these advancements could pave the way for more effective therapies for genetic disorders.

Why it matters

Potential benefit: If successful, this research could lead to safer and more effective gene editing therapies for patients with genetic disorders like sickle cell disease.

How similar studies have gone: Previous research has shown promise in using CRISPR technology for gene editing, indicating that this approach could lead to significant advancements in treatment.

Where this research is happening

  • Genetobe INC.
    Ann Arbor, United States
    Active

Researchers

  • Principal investigator: Hou, Zhonggang — Genetobe INC.
  • Study coordinator: Hou, Zhonggang

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  • Gene editing therapy for a genetic disorder causing hearing and vision loss

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About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
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Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.