This federal Project Grant award, valued at $253,167.00 and issued on August 23, 2024 by the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research), aims to develop a non-viral gene therapy approach to prevent fertility impairments in women affected by classic galactosemia, a rare genetic disorder. The key objectives of this research project are to: 1) Develop a nanoparticle-based gene therapy that can deliver episomal GALT cDNA to granulosa cells to restore enzyme activity, and 2) Determine the optimal developmental stage for intervention to prevent ovarian damage. This will involve using the zebrafish model of classic galactosemia to study folliculogenesis, gene expression patterns, and histological changes at different developmental stages. The research team includes sub-awardees from the Cleveland Clinic Lerner College of Medicine, Boston Children's Hospital, and Maastricht University, who will contribute specialized expertise in areas such as advanced gene delivery, single-cell RNA sequencing, and animal model studies.
Mod # | Description | Reason For Modification | Federal Obligation (Click to sort descending) | Date (Click to sort ascending) |
|---|---|---|---|---|
| Not listed | $253.2k | 8/23/24 |
Grant Number | Description | Subgrantee | Prime Award | Dollars Obligated (Click to sort descending) | Updated At (Click to sort ascending) |
|---|---|---|---|---|---|
1006816403CCLCMS | Cleveland Clinic Lerner College Of Medicine Of Case Western Reserve University | Project Grant R21HD113931 | $50.1k | 1/2/25 | |
1006816402BCHS | The Children's Hospital Corporation | Project Grant R21HD113931 | $42.8k | 11/22/24 | |
1006816401UMS | Universiteit Maastricht | Project Grant R21HD113931 | $74.3k | 10/30/24 |