ArticleStem cell research & therapy2021
Development, characterization, and hematopoietic differentiation of Griscelli syndrome type 2 induced pluripotent stem cells.
Article in Stem cell research & therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed, 9 citations in OpenAlex.
- Establishment, Characterization, and Cryopreservation of Feather Follicle Fibroblast Lines From Hyacinth Macaw (Anodorhynchus hyacinthinus).Cell biology international · 2025Article
- Endocannabinoid system upregulates the enrichment and differentiation of human iPSC- derived spermatogonial stem cells via CB2R agonism.Biological research · 2025Article
- The Role of Rab GTPases in the development of genetic and malignant diseases.Molecular and cellular biochemistry · 2024Review
- The occurrence and development of induced pluripotent stem cells.Frontiers in genetics · 2024Review
- Correction of Griscelli Syndrome Type 2 causing mutations in theTurkish journal of biology = Turk biyoloji dergisi · 2024Article
- Generation and Hematopoietic Differentiation of Mesenchymal Stromal/Stem Cell-Derived Induced Pluripotent Stem Cell Lines for Disease Modeling of Hematopoietic and Immunological Diseases.Methods in molecular biology (Clifton, N.J.) · 2022Article
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Authors and funding
10 authors at 2 institutions in 1 country.
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Abstract
backgroundGriscelli syndrome type 2 (GS-2) is a rare, autosomal recessive immune deficiency syndrome caused by a mutation in the RAB27A gene, which results in the absence of a protein involved in vesicle trafficking and consequent loss of function of in particular cytotoxic T and NK cells. Induced pluripotent stem cells (iPSC) express genes associated with pluripotency, have the capacity for infinite expansion, and can differentiate into cells from all three germ layers. They can be induced using integrative or non-integrative systems for transfer of the Oct4, Sox2, Klf4, and cMyc (OSKM) transcription factors. To better understand the pathophysiology of GS-2 and to test novel treatment options, there is a need for an in vitro model of GS-2.
methodsHere, we generated iPSCs from 3 different GS-2 patients using lentiviral vectors. The iPSCs were characterized using flow cytometry and RT-PCR and tested for the expression of pluripotency markers. In vivo differentiation to cells from all three germlines was tested using a teratoma assay. In vitro differentiation of GS-2 iPSCs into hematopoietic stem and progenitor cells was done using Op9 feeder layers and specified media.
resultsAll GS-2 iPSC clones displayed a normal karyotype (46XX or 46XY) and were shown to express the same RAB27A gene mutation that was present in the original somatic donor cells. GS-2 iPSCs expressed SSEA1, SSEA4, TRA-1-60, TRA-1-81, and OCT4 proteins, and SOX2, NANOG, and OCT4 expression were confirmed by RT-PCR. Differentiation capacity into cells from all three germ layers was confirmed using the teratoma assay. GS-2 iPSCs showed the capacity to differentiate into cells of the hematopoietic lineage.
conclusionsUsing the lentiviral transfer of OSKM, we were able to generate different iPSC clones from 3 GS-2 patients. These cells can be used in future studies for the development of novel treatment options and to study the pathophysiology of GS-2 disease.
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