Developing personalized gene therapies for rare genetic diseases in children
Providing ethical guidance for the development of individualized genomic medicine as rare as n-of-1
This study is working on personalized treatments for kids with rare genetic diseases that don’t have any approved medicines yet, using special tools to target their unique genetic needs and ensuring that these treatments are safe and ethical.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10888228 on NIH RePORTER |
What this research studies
This research focuses on creating individualized gene-targeted treatments for children with rare genetic diseases, known as orphan diseases, which currently have no approved therapies. The approach involves designing specific antisense oligonucleotides (ASOs) that can target unique genetic variants in individual patients. By addressing the ethical, legal, and social implications of these personalized treatments, the research aims to provide a framework for safely and effectively delivering these therapies to children who need them most.
Who could benefit from this research
Good fit: Ideal candidates for this research are children under 11 years old diagnosed with rare genetic diseases that currently lack approved treatments.
Not a fit: Patients with common genetic disorders or those over the age of 11 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to effective, personalized treatments for children suffering from rare genetic disorders.
How similar studies have performed: Other research has shown promise in developing individualized therapies for rare diseases, particularly using antisense oligonucleotides, indicating a growing field with potential for success.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Yu, Timothy Wei-Wen — Boston Children's Hospital
- Study coordinator: Yu, Timothy Wei-Wen
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.