Personalized prime editing as a platform for hepatic inborn errors of metabolism — Rebecca Clare Ahrens-Nicklas (CHILDREN'S HOSP O
PROJECT SUMMARY Inborn errors of metabolism (IEMs) are rare, devasting disorders arising from pathogenic variants in genes encoding enzymes of key biochemical pathways. The liver plays an important ro
| Project title | Personalized prime editing as a platform for hepatic inborn errors of metabolism |
|---|---|
| Project number | 1U01TR005355-01 |
| PI name | Rebecca Clare Ahrens-Nicklas |
| Institution | CHILDREN'S HOSP OF PHILADELPHIA |
| Funding mechanism | U01 |
| Agency | NCATS |
| Award amount | $2.75M |
| Fiscal year | 2024 |
| Project start | 2024-09-20T00:00:00 |
| Project end | 2028-08-31T00:00:00 |
| Abstract | PROJECT SUMMARY Inborn errors of metabolism (IEMs) are rare, devasting disorders arising from pathogenic variants in genes encoding enzymes of key biochemical pathways. The liver plays an important ro |
| Key terms | <21+ years old><AAV delivered><AAV delivery><AAV-based delivery><AAV-based viral delivery><AAV-media |
$799/mo
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