α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment

Abstract: Cytoskeletal remodeling and mitochondrial bioenergetics play important roles in thrombocytopoiesis and platelet function. Recently, α-actinin-1 mutations have been reported in patients with congenital macrothrombocytopenia. However, the role and underlying mechanism of α-actinin-1 in throm...

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Main Authors: Xiangjie Lin, Hanchen Gao, Min Xin, Jian Huang, Xia Li, Yutong Zhou, Keyu Lv, Xin Huang, Jinghan Wang, Yulan Zhou, Dawei Cui, Chao Fang, Lanlan Wu, Xiaofeng Shi, Zhixin Ma, Yu Qian, Hongyan Tong, Jing Dai, Jie Jin, Jiansong Huang
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925000217
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author Xiangjie Lin
Hanchen Gao
Min Xin
Jian Huang
Xia Li
Yutong Zhou
Keyu Lv
Xin Huang
Jinghan Wang
Yulan Zhou
Dawei Cui
Chao Fang
Lanlan Wu
Xiaofeng Shi
Zhixin Ma
Yu Qian
Hongyan Tong
Jing Dai
Jie Jin
Jiansong Huang
author_facet Xiangjie Lin
Hanchen Gao
Min Xin
Jian Huang
Xia Li
Yutong Zhou
Keyu Lv
Xin Huang
Jinghan Wang
Yulan Zhou
Dawei Cui
Chao Fang
Lanlan Wu
Xiaofeng Shi
Zhixin Ma
Yu Qian
Hongyan Tong
Jing Dai
Jie Jin
Jiansong Huang
author_sort Xiangjie Lin
collection DOAJ
description Abstract: Cytoskeletal remodeling and mitochondrial bioenergetics play important roles in thrombocytopoiesis and platelet function. Recently, α-actinin-1 mutations have been reported in patients with congenital macrothrombocytopenia. However, the role and underlying mechanism of α-actinin-1 in thrombocytopoiesis and platelet function remain elusive. Using megakaryocyte (MK)–specific α-actinin-1 knockout (KO; PF4-Actn1−/−) mice, we demonstrated that PF4-Actn1−/− mice exhibited reduced platelet counts. The decreased platelet number in PF4-Actn1−/− mice was due to defects in thrombocytopoiesis. Hematoxylin and eosin staining and flow cytometry revealed a decrease in the number of MKs in the bone marrow of PF4-Actn1−/− mice. The absence of α-actinin-1 increased the proportion of 2 N-4 N MKs and decreased the proportion of 8 N-32 N MKs. Colony-forming unit–MK colony formation, the ratio of proplatelet formation–bearing MKs, and MK migration in response to stromal cell–derived factor-1 signaling were inhibited in PF4-Actn1−/− mice. Platelet spreading, clot retraction, aggregation, integrin αIIbβ3 activation, and CD62P exposure in response to various agonists were decreased in PF4-Actn1−/− platelets. Notably, PF4-Actn1−/− platelets inhibited calcium mobilization, reactive oxygen species (ROS) generation, and actin polymerization in response to collagen and thrombin. Furthermore, the PF4-Actn1−/− mice exhibited impaired hemostasis and thrombosis. Mechanistically, proteomic analysis of low-ploidy (2-4 N) and high-ploidy (≥8 N) PF4-Actn1−/− MKs revealed that α-actinin-1 deletion reduced platelet activation and mitochondrial function. PF4-Actn1−/− platelets and Actn1 KO 293T cells exhibited reduced mitochondrial membrane potential, mitochondrial ROS generation, mitochondrial calcium mobilization, and mitochondrial bioenergetics. Overall, in this study, we report that mice with α-actinin-1 deficiency in MKs exhibit low platelet count and impaired platelet function, thrombosis, and mitochondrial bioenergetics.
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spelling doaj-art-cc5ee67605014b0cbbeab96c2e00e1462025-08-20T02:02:05ZengElsevierBlood Advances2473-95292025-03-01951185120110.1182/bloodadvances.2024014805α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairmentXiangjie Lin0Hanchen Gao1Min Xin2Jian Huang3Xia Li4Yutong Zhou5Keyu Lv6Xin Huang7Jinghan Wang8Yulan Zhou9Dawei Cui10Chao Fang11Lanlan Wu12Xiaofeng Shi13Zhixin Ma14Yu Qian15Hongyan Tong16Jing Dai17Jie Jin18Jiansong Huang19Department of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Laboratory Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Pharmacology, School of Basic Medicine, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, The First Affiliated Hospital of Nanchang University, Nanchang, ChinaDepartment of Blood Transfusion, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Pharmacology, School of Basic Medicine, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, ChinaDepartment of Emergency Medicine, The Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, The Second Affiliated Hospital of Nanjing Medical University, Nanjing, ChinaClinical Prenatal Diagnosis Center, Key Laboratory of Reproductive Genetics, Women's Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, ChinaDepartment of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Hongyan Tong, Department of Hematology, The First Affiliated Hospital, Zhejiang University School of Medicine, 79 Qingchun Rd, Hangzhou 310003, China;Department of Laboratory Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China; Jing Dai, Department of Laboratory Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, No. 197 Ruijin Er Rd, Shanghai 200025, China;Department of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Cancer Center, Zhejiang University, Hangzhou, China; Zhejiang Provincial Clinical Research Center for Hematological Disorders, Hangzhou, China; Jie Jin, Department of Hematology, The First Affiliated Hospital, Zhejiang University School of Medicine, 79 Qingchun Rd, Hangzhou 310003, China;Department of Hematology, Zhejiang Key Laboratory for Precision Diagnosis and Treatment of Hematological Malignancies, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Correspondence: Jiansong Huang, Department of Hematology, The First Affiliated Hospital, Zhejiang University School of Medicine, 79 Qingchun Rd, Hangzhou 310003, China;Abstract: Cytoskeletal remodeling and mitochondrial bioenergetics play important roles in thrombocytopoiesis and platelet function. Recently, α-actinin-1 mutations have been reported in patients with congenital macrothrombocytopenia. However, the role and underlying mechanism of α-actinin-1 in thrombocytopoiesis and platelet function remain elusive. Using megakaryocyte (MK)–specific α-actinin-1 knockout (KO; PF4-Actn1−/−) mice, we demonstrated that PF4-Actn1−/− mice exhibited reduced platelet counts. The decreased platelet number in PF4-Actn1−/− mice was due to defects in thrombocytopoiesis. Hematoxylin and eosin staining and flow cytometry revealed a decrease in the number of MKs in the bone marrow of PF4-Actn1−/− mice. The absence of α-actinin-1 increased the proportion of 2 N-4 N MKs and decreased the proportion of 8 N-32 N MKs. Colony-forming unit–MK colony formation, the ratio of proplatelet formation–bearing MKs, and MK migration in response to stromal cell–derived factor-1 signaling were inhibited in PF4-Actn1−/− mice. Platelet spreading, clot retraction, aggregation, integrin αIIbβ3 activation, and CD62P exposure in response to various agonists were decreased in PF4-Actn1−/− platelets. Notably, PF4-Actn1−/− platelets inhibited calcium mobilization, reactive oxygen species (ROS) generation, and actin polymerization in response to collagen and thrombin. Furthermore, the PF4-Actn1−/− mice exhibited impaired hemostasis and thrombosis. Mechanistically, proteomic analysis of low-ploidy (2-4 N) and high-ploidy (≥8 N) PF4-Actn1−/− MKs revealed that α-actinin-1 deletion reduced platelet activation and mitochondrial function. PF4-Actn1−/− platelets and Actn1 KO 293T cells exhibited reduced mitochondrial membrane potential, mitochondrial ROS generation, mitochondrial calcium mobilization, and mitochondrial bioenergetics. Overall, in this study, we report that mice with α-actinin-1 deficiency in MKs exhibit low platelet count and impaired platelet function, thrombosis, and mitochondrial bioenergetics.http://www.sciencedirect.com/science/article/pii/S2473952925000217
spellingShingle Xiangjie Lin
Hanchen Gao
Min Xin
Jian Huang
Xia Li
Yutong Zhou
Keyu Lv
Xin Huang
Jinghan Wang
Yulan Zhou
Dawei Cui
Chao Fang
Lanlan Wu
Xiaofeng Shi
Zhixin Ma
Yu Qian
Hongyan Tong
Jing Dai
Jie Jin
Jiansong Huang
α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
Blood Advances
title α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
title_full α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
title_fullStr α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
title_full_unstemmed α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
title_short α-Actinin-1 deficiency in megakaryocytes causes low platelet count, platelet dysfunction, and mitochondrial impairment
title_sort α actinin 1 deficiency in megakaryocytes causes low platelet count platelet dysfunction and mitochondrial impairment
url http://www.sciencedirect.com/science/article/pii/S2473952925000217
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