Human Gaucher’s Type 1 iPSCs
Our Gaucher’s Type 1 iPSC line is derived from a 1-year-old male donor of African American descent diagnosed with Gaucher’s Type 1, the most common lysosomal storage disorder. A defective glucocerebrosidase (GCase) enzyme causes this disease by impairing the breakdown of glucocerebroside, a sphingolipid normally processed within lysosomes. As a result, glucocerebroside accumulates in tissues such as the spleen, liver, kidneys, lungs, brain, and bone marrow, leading to progressive neurological complications, hepatosplenomegaly, anemia, and bone abnormalities [i].
This iPSC line provides a valuable platform for studying Gaucher disease mechanisms, screening potential treatments, and developing gene therapy strategies. Using our patented episomal reprogramming method, we have generated a high-fidelity, non-integrating iPSC line that maintains genomic integrity while ensuring consistent, efficient reprogramming. We recommend culturing this cell line with our Human iPSC Growth Media (CET-MR1001-K).
We enhance clinical safety by excluding Myc and Lin28 transcription factors from reprogramming, as they are linked to neoplastic formation [ii]. This approach lowers the clinical risk profile of downstream differentiated cells, making this iPSC line an ideal model for Gaucher disease research, drug discovery, and regenerative medicine.
Key Features & Quality Control:
- Glucocerebrosidase-deficient iPSC line validated for pluripotency
- Non-integrating episomal reprogramming for genomic stability
- Mycoplasma-free and pathogen-free certification
- Cryopreserved at low passage to ensure high post-thaw viability
Applications of Gaucher’s Type 1 iPSCs:
- Disease modeling for lysosomal storage disorders
- Drug screening for glucocerebrosidase enzyme replacement and chaperone therapy
- Gene therapy development for precision medicine applications
- Differentiation into macrophages, hepatocytes, and neuronal models
Specifications:
- Cell Type: Human induced pluripotent stem cells (iPSCs)
- Donor Information: 1-year-old male, African American descent
- Reprogramming Method: Non-integrating episomal DNA
- Mutation: GBA1 gene mutation (variant details available upon request)
- Culture Conditions: Feeder-free, compatible with standard iPSC growth media
- Storage & Shipping: Cryopreserved, shipped on dry ice
Each vial contains ~500,000 cryopreserved cells, providing a robust, reproducible model for lysosomal storage disease research and therapeutic advancements.
| Documents & Links for Human Gaucher's Type 1 iPS Cells | |
| Datasheet | Human Gaucher's Type 1 iPS Cells Datasheet |
| Vendor Page | Human Gaucher's Type 1 iPS Cells at Cellular Engineering Technologies |
| Documents & Links for Human Gaucher's Type 1 iPS Cells | |
| Datasheet | Human Gaucher's Type 1 iPS Cells Datasheet |
| Vendor Page | Human Gaucher's Type 1 iPS Cells |