| Gene symbol | GBA1 | Synonyms | GBA, GCB, GLUC | Type of gene | protein-coding |
| Chromosome | 1 | Map location | 1q22 | dbXrefs | |
| Gene perturbation-related omics dataset | PerturbAtlas | ||||
| Description | glucosylceramidase beta 1 | ||||
| GTO ID | GTC1166 |
| Trial ID | NCT00004294 |
| Disease | Gaucher's Disease |
| Altered gene | GBA1 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | GBA LV-modified PBSCs |
| Phase | Phase1 |
| Recruitment status | Completed |
| Title | Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease |
| Year | 1999 |
| Country | United States |
| Company sponsor | National Center for Research Resources (NCRR) |
| Other ID(s) | 199/11727|UPITTS-GAUCHER |
| Vector information | |||||
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| Cohort 1 | |||||||||
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