Stem cell treatment & Gaucher disease

Written from the studies on PubMed, searched on 2026. nov. 11.. Every number below comes from a study listed under Sources.

What people read

Posts online say that Stem cell treatment helps with Gaucher disease.

What has been studied

• 1In people
• 2In animals
• 15Only in the lab

What the studies found

!Stem cell treatment has been studied in people with Gaucher disease, but not in randomised trials.

We found 49 published papers on Stem cell treatment and Gaucher disease. They were published between 1991 and 2026.

1 of them are studies in people (0 randomised trials), 11 are reviews, 2 are studies in animals and 15 are studies only in the lab.

The most important ones are listed under Sources.

Sources

1. Retroviral mediated transfer of the cDNA for human glucocerebrosidase into hematopoietic stem cells of patients with Gaucher disease. A phase I study.1996 · Hum Gene Ther · Study in people
2. Current opinion on pluripotent stem cell technology in Gaucher's disease: challenges and future prospects.2025 · Cytotechnology · Review
3. Successful Treatment of Gaucher Disease With Matched Sibling Hematopoietic Stem Cell Transplantation: A Case Report and Literature Review.2021 · J Pediatr Hematol Oncol · Review
4. Hematopoietic stem cell transplantation for Gaucher disease.2017 · Cochrane Database Syst Rev · Review
5. Induced Pluripotent Stem Cell Modeling of Gaucher's Disease: What Have We Learned?2017 · Int J Mol Sci · Review
6. [Treatment of Gaucher disease with allogeneic hematopoietic stem cell transplantation: report of three cases and review of literatures].2015 · Zhonghua Er Ke Za Zhi · Review
7. Gauchers disease--a reappraisal of hematopoietic stem cell transplantation.2013 · Pediatr Hematol Oncol · Review
8. A prospective study of bone marrow hematopoietic and mesenchymal stem cells in type 1 Gaucher disease patients.2013 · PLoS One · In animals
9. Expression of human glucocerebrosidase following retroviral vector-mediated transduction of murine hematopoietic stem cells.1991 · Bone Marrow Transplant · In animals
10. Establishment of a human induced pluripotent stem cell line (PNUSCRi003-A) from a patient with Gaucher disease carrying compound heterozygous p.Arg87Trp and p.Arg296Gln variants in the GBA1 gene.2026 · Stem Cell Res · In the lab
11. Evaluation of Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Siblings with Gaucher Disease Discordant for Parkinsonism.2025 · Mov Disord · In the lab
12. Establishment of MUi030-A: A human induced pluripotent stem cell line carrying homozygous L444P mutation in the GBA1 gene to study type-3 Gaucher disease.2023 · Stem Cell Res · In the lab
13. A generation of human induced pluripotent stem cell line (MUi031-A) from a type-3 Gaucher disease patient carrying homozygous mutation on GBA1 gene.2022 · Stem Cell Res · In the lab
14. Correction to "Evaluation of Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Siblings with Gaucher Disease Discordant for Parkinsonism".2026 · Mov Disord · Other paper
15. Autologous genome-edited hematopoietic stem cells correct Gaucher disease and establish a platform for clinical translation.2025 · Res Sq · Other paper
16. Hematopoietic stem cell transplantation or enzyme replacement therapy in Gaucher disease type 3.2024 · Mol Genet Metab · Other paper
17. CRISPR Correction of the GBA Mutation in Human-Induced Pluripotent Stem Cells Restores Normal Function to Gaucher Macrophages and Increases Their Susceptibility to Mycobacterium tuberculosis.2023 · J Infect Dis · Other paper

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