ArticleStem cell reviews and reports2026
Assessment of Potential Side Effects Related To RAB27A Gene Therapy in Stem Cells.
Article in Stem cell reviews and reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
Abstract
RAB27A plays an essential role in the regulation of exocytosis and intracellular vesicle trafficking. Loss-of-function mutations in the RAB27A gene cause dysfunctional immune cells and Griscelli Syndrome Type 2 (GS-2), whereas upregulation of RAB27A in cancer cells is associated with a worse prognosis and increased metastasis. Here, we wanted to assess the potential side effects of overexpression of RAB27A in different types of healthy stem cells as preparation for the development of gene therapy for GS-2. Bone marrow mesenchymal stem cells (BM-MSCs) were obtained from GS-2 patients and healthy donors. Healthy murine bone marrow-derived and human cord blood-derived hematopoietic stem/progenitor cells (HSPCs) were transduced with different lentiviral vectors carrying a codon-optimized RAB27A (RAB27Aco) transgene. Cells were used for in vitro functional assays and assessed using flow cytometry, Western Blot and RT-PCR. In vivo transplantation assays in mice were used to assess the effect of RAB27A on stem cell function. Engraftment was assessed using flow cytometry, sections of BM-MSC injection sites were analyzed using histological staining. Overexpression of RAB27A resulted in phenotypic changes in BM-MSCs and decreased colony-forming capacity of HSPCs. Transplantation of RAB27A + stem cells was not associated with any tumorigenesis. Despite high expression of RAB27A in HSPCs before transplantation, RAB27A levels in peripheral blood, bone marrow, and spleen cells remained low, indicating overexpression of RAB27A may have affected the long-term reconstitution potential. Development of gene therapy for GS-2 may require fine-tuning of RAB27A expression but is not likely to be complicated by RAB27A-induced tumorigenesis.
Indexed as
Identifiers
41075149What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.