An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets

PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions that lead to overgrowth of various body parts resulting from activating variants in PIK3CA. The absence of ideal cell models significantly impedes progress in PROS research. In this study, we focused on facial infiltrating...

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Main Authors: Bin Sun, Hongrui Chen, Wei Gao, Yunqi Li, Chen Hua, Xiaoxi Lin
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Biochemistry and Biophysics Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2405580824002334
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author Bin Sun
Hongrui Chen
Wei Gao
Yunqi Li
Chen Hua
Xiaoxi Lin
author_facet Bin Sun
Hongrui Chen
Wei Gao
Yunqi Li
Chen Hua
Xiaoxi Lin
author_sort Bin Sun
collection DOAJ
description PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions that lead to overgrowth of various body parts resulting from activating variants in PIK3CA. The absence of ideal cell models significantly impedes progress in PROS research. In this study, we focused on facial infiltrating lipomatosis (FIL) (A disorder within PROS) and aimed to establish and characterize an immortalized PROS cell line. Primary adipose-derived stem cells of FIL were immortal-ized through the transfection of simian virus 40 large T antigen (SV40LT). No significant mor-phological differences were observed in immortalized FIL-ADSCs (Im FIL-ADSCs). Im FIL-ADSCs expressed original mesenchymal surface markers, confirmed by flow cytometry. It harbored PIK3CA mutation and an increased level of PI3K/AKT activation, revealed by sanger sequencing and Western blot respectively. Karyotype analysis revealed a stable chromosome in Im FIL-ADSCs. Higher adipogenic potential and lower osteogenic differentiation properties were de-tected in Im FIL-ADSCs. The proliferative potential of Im FIL-ADSCs increased, whereas malig-nant transformation was not observed in the tumorigenesis assay. Moreover, RNA sequencing further elucidated the role of the transcription factor E2F1 in Im FIL-ADSCs. Drug screening unveiled that STAT3, HSP, EGFR, and NF-kB might be potential therapeutic targets for FIL. This study provided a valuable cellular resource for exploring the underlying pathogenic mechanisms and developing new targeted therapeutic options for PROS.
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spelling doaj-art-3ddeb2d95ea84fea87d7212381c1ebfa2025-08-20T02:30:32ZengElsevierBiochemistry and Biophysics Reports2405-58082024-12-014010186910.1016/j.bbrep.2024.101869An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targetsBin Sun0Hongrui Chen1Wei Gao2Yunqi Li3Chen Hua4Xiaoxi Lin5Department of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, ChinaDepartment of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, ChinaDepartment of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, ChinaShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, ChinaDepartment of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China; Corresponding author. Department of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, PR China, 639 Zhizaoju Road, Shanghai, 200011, China.Department of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China; Corresponding author. Department of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, PR China 639 Zhizaoju Road, Shanghai, 200011, China.PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions that lead to overgrowth of various body parts resulting from activating variants in PIK3CA. The absence of ideal cell models significantly impedes progress in PROS research. In this study, we focused on facial infiltrating lipomatosis (FIL) (A disorder within PROS) and aimed to establish and characterize an immortalized PROS cell line. Primary adipose-derived stem cells of FIL were immortal-ized through the transfection of simian virus 40 large T antigen (SV40LT). No significant mor-phological differences were observed in immortalized FIL-ADSCs (Im FIL-ADSCs). Im FIL-ADSCs expressed original mesenchymal surface markers, confirmed by flow cytometry. It harbored PIK3CA mutation and an increased level of PI3K/AKT activation, revealed by sanger sequencing and Western blot respectively. Karyotype analysis revealed a stable chromosome in Im FIL-ADSCs. Higher adipogenic potential and lower osteogenic differentiation properties were de-tected in Im FIL-ADSCs. The proliferative potential of Im FIL-ADSCs increased, whereas malig-nant transformation was not observed in the tumorigenesis assay. Moreover, RNA sequencing further elucidated the role of the transcription factor E2F1 in Im FIL-ADSCs. Drug screening unveiled that STAT3, HSP, EGFR, and NF-kB might be potential therapeutic targets for FIL. This study provided a valuable cellular resource for exploring the underlying pathogenic mechanisms and developing new targeted therapeutic options for PROS.http://www.sciencedirect.com/science/article/pii/S2405580824002334PIK3CA-Related overgrowth spectrumFacial infiltrating lipomatosisImmortalizationADSCsTarget therapy
spellingShingle Bin Sun
Hongrui Chen
Wei Gao
Yunqi Li
Chen Hua
Xiaoxi Lin
An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
Biochemistry and Biophysics Reports
PIK3CA-Related overgrowth spectrum
Facial infiltrating lipomatosis
Immortalization
ADSCs
Target therapy
title An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
title_full An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
title_fullStr An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
title_full_unstemmed An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
title_short An immortalized adipose-derived stem cells line from the PIK3CA-related overgrowth spectrum: Unveiling novel therapeutic targets
title_sort immortalized adipose derived stem cells line from the pik3ca related overgrowth spectrum unveiling novel therapeutic targets
topic PIK3CA-Related overgrowth spectrum
Facial infiltrating lipomatosis
Immortalization
ADSCs
Target therapy
url http://www.sciencedirect.com/science/article/pii/S2405580824002334
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