Targeted DOK7 gene therapy for Congenital Myasthenic Syndromes — Patricio Sepulveda (AMPLO BIOTECHNOLOGY, INC.)
PROJECT SUMMARY Congenital Myasthenic Syndromes (CMS) are a group of genetically and phenotypically heterogeneous, neuromuscular transmission disorders characterized by muscle weakness (myasthenia) th
| Project title | Targeted DOK7 gene therapy for Congenital Myasthenic Syndromes |
|---|---|
| Project number | 2R44NS124351-04 |
| PI name | Patricio Sepulveda |
| Institution | AMPLO BIOTECHNOLOGY, INC. |
| Funding mechanism | R44 |
| Agency | NINDS |
| Award amount | $3.00M |
| Fiscal year | 2024 |
| Project start | 2021-09-15T00:00:00 |
| Project end | 2026-08-31T00:00:00 |
| Abstract | PROJECT SUMMARY Congenital Myasthenic Syndromes (CMS) are a group of genetically and phenotypically heterogeneous, neuromuscular transmission disorders characterized by muscle weakness (myasthenia) th |
| Key terms | <0-11 years old><21+ years old><AAV vector><AAV-based vector><Acetylcholine><Acetylcholine Hydrolase |
$799/mo
Try GrantRadar →