Aims: The purpose of this study was to determine the functional recovery and protein regulation by transplanted induced pluripotent stem cells in a rat model of Huntington's disease (HD).
Methods: In a quinolinic acid-induced rat model of striatal degeneration, induced pluripotent stem cells were transplanted into the ipsilateral lateral ventricle 10 days after the quinolinic acid injection. At 8 weeks after transplantation, fluorodeoxyglucose-PET/CT scan and balance-beam test were performed to evaluate the functional recovery of experimental rats. In addition, immunofluorescence and protein array analysis were used to investigate the regulation of stimulated protein expression in the striatum.
Results: At 8 weeks after induced pluripotent stem cell transplantation, motor function was improved in comparison with the quinolinic acid-treated rats. High fluorodeoxyglucose accumulation in the injured striatum was also observed by PET/CT scans. In addition, immunofluorescence analysis demonstrated that implanted cells migrated from the lateral ventricle into the lesioned striatum and differentiated into striatal projection neurons. Array analysis showed a significant upregulation of GFR (Glial cell line-derived neurotrophic factor receptor) alpha-1, Adiponectin/Acrp30, basic-fibroblast growth factors, MIP-1 (Macrophage-inflammatory protein) alpha and leptin, as well as downregulation of cytokine-induced neutrophil chemoattractant-3 in striatum after transplantatation of induced pluripotent stem cells in comparison with the quinolinic acid -treated rats.
Conclusions: The findings in this work indicate that transplantation of induced pluripotent stem cells is a promising therapeutic candidate for HD.
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http://dx.doi.org/10.1111/nan.12315 | DOI Listing |
Stem Cell Res
December 2024
Emergency and Critical Care Department, University of Health and Rehabilitation Sciences (Qingdao Central Hospital), Qingdao 266042, China. Electronic address:
A human induced pluripotent stem cell (iPSC) line was generated from patient with Kennedy Disease (KD), who carried the CAG repeat expansion mutation in AR gene. Peripheral blood mononuclear cells (PBMCs) were reprogrammed using non-integrating delivery of KFL4, OCT4, SOX2, BCL-XL and c-MYC. The iPSC line expresses pluripotency markers, displays a normal karyotype, and is capable of differentiate into three germ layers in vitro.
View Article and Find Full Text PDFStem Cell Rev Rep
January 2025
Department of Biosciences and Bioengineering, Indian Institute of Technology Guwahati, Guwahati, Assam, India.
Background: Undifferentiated embryonic cell transcription factor 1 (UTF1) is predominantly expressed in pluripotent stem cells and plays a vital role in embryonic development and pluripotency maintenance. Despite its established importance in murine models, the role of UTF1 on human induced pluripotent stem cells (iPSCs) has not been comprehensively studied.
Methods: This study utilized CRISPR/Cas9 gene editing to create UTF1 knockout in human fibroblasts and iPSCs.
Autosomal-recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is an early-onset neurodegenerative disease caused by mutations in the SACS gene. The first two mutations were identified in French Canadian populations 20 years ago. The disease is now known as one of the most frequent recessive ataxias worldwide.
View Article and Find Full Text PDFStem Cell Reports
December 2024
Department of Cardio Metabolic Diseases Research, Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach, Germany. Electronic address:
Complement factor H (CFH) common genetic variants have been associated with age-related macular degeneration (AMD). While most previous in vitro RPE studies focused on the common p.His402Tyr CFH variant, we characterized rare CFH variants that are highly penetrant for AMD using induced pluripotent stem-cell-derived retinal pigment epithelium (iPSC-RPE).
View Article and Find Full Text PDFCell Stem Cell
January 2025
Department of Chemical and Biological Engineering, University of Wisconsin-Madison, Madison, WI, USA. Electronic address:
Tissue-engineered vascular conduits (TEVCs) are a promising blood vessel replacement. In a recent publication in Cell Stem Cell, Park et al. developed TEVCs comprised of decellularized human umbilical arteries lined with shear-trained, human induced pluripotent stem cell (hiPSC)-derived endothelial cells (ECs) that resisted thrombosis and exhibited patency upon grafting into the rat inferior vena cava (IVC).
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