Developing advanced tools for genome editing
Scalable Development of Custom Genome Editing Technologies
['FUNDING_OTHER'] · MASSACHUSETTS GENERAL HOSPITAL · NIH-10472972
This study is working on making gene editing tools better so they can help treat more genetic disorders by creating improved versions of the CRISPR system, which could lead to more effective and personalized treatments for patients like you.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MASSACHUSETTS GENERAL HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-10472972 on ClinicalTrials.gov |
What this research studies
This research focuses on improving genome editing technologies, specifically by enhancing the CRISPR-Cas9 system to target a wider range of genetic mutations. The approach involves using directed evolution to create new versions of the CRISPR enzyme that can recognize different DNA sequences, which is crucial for treating various genetic disorders. By overcoming the limitations of existing genome editing methods, this research aims to make genetic therapies more effective and accessible. Patients may benefit from more precise and versatile treatments for genetic conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders caused by mutations that are currently difficult to target with existing genome editing technologies.
Not a fit: Patients with genetic conditions that do not involve mutations suitable for CRISPR editing may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for a variety of genetic disorders by enabling precise editing of previously untargetable mutations.
How similar studies have performed: Other research has shown promise in enhancing genome editing technologies, but this specific approach of directed evolution for CRISPR is innovative and largely untested.
Where this research is happening
BOSTON, UNITED STATES
- MASSACHUSETTS GENERAL HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: KLEINSTIVER, BENJAMIN PETER — MASSACHUSETTS GENERAL HOSPITAL
- Study coordinator: KLEINSTIVER, BENJAMIN PETER
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.
Conditions: disease-causing mutation