Human-induced pluripotent stem cell approaches to model inborn and acquired metabolic heart diseases

被引:10
作者
Chanana, Anita M. [1 ,2 ]
Rhee, June-Wha [1 ,2 ]
Wu, Joseph C. [1 ,2 ,3 ,4 ]
机构
[1] Stanford Univ, Sch Med, Stanford Cardiovasc Inst, Stanford, CA 94305 USA
[2] Stanford Univ, Sch Med, Dept Med, Div Cardiol, Stanford, CA 94305 USA
[3] Stanford Univ, Sch Med, Dept Radiol, Stanford, CA 94305 USA
[4] Stanford Univ, Sch Med, Inst Stem Cell Biol & Regenerat Med, Stanford, CA 94305 USA
基金
美国国家卫生研究院;
关键词
cardiomyocytes; induced pluripotent stem cells; metabolic heart disease; POMPE DISEASE; BARTH-SYNDROME; CARDIOMYOPATHY; PATIENT; FIBROBLASTS; GENERATION; PHENOTYPE;
D O I
10.1097/HCO.0000000000000277
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Purpose of review The article provides an overview of advances in the induced pluripotent stem cell field to model cardiomyopathies of inherited inborn errors of metabolism and acquired metabolic syndromes in vitro. Recent findings Several inborn errors of metabolism have been studied using disease in a dish' models, including Pompe disease, Danon disease, Fabry disease, and Barth syndrome. Disease phenotypes of complex metabolic syndromes, such as diabetes mellitus and aldehyde dehydrogenase 2 deficiency, have also been observed. Summary Differentiation of patient and disease-specific induced pluripotent stem cell-derived cardiomyocytes has provided the capacity to model deleterious cardiometabolic diseases to understand molecular mechanisms, perform drug screens, and identify novel drug targets.
引用
收藏
页码:266 / 274
页数:9
相关论文
共 49 条
  • [1] Identification of a New Modulator of the Intercalated Disc in a Zebrafish Model of Arrhythmogenic Cardiomyopathy
    Asimaki, Angeliki
    Kapoor, Sudhir
    Plovie, Eva
    Arndt, Anne Karin
    Adams, Edward
    Liu, ZhenZhen
    James, Cynthia A.
    Judge, Daniel P.
    Calkins, Hugh
    Churko, Jared
    Wu, Joseph C.
    MacRae, Calum A.
    Kleber, Andre G.
    Saffitz, Jeffrey E.
    [J]. SCIENCE TRANSLATIONAL MEDICINE, 2014, 6 (240)
  • [2] Natural history of Danon disease
    Boucek, Dana
    Jirikowic, Jean
    Taylor, Matthew
    [J]. GENETICS IN MEDICINE, 2011, 13 (06) : 563 - 568
  • [3] Diabetic cardiomyopathy revisited
    Boudina, Sihem
    Abel, E. Dale
    [J]. CIRCULATION, 2007, 115 (25) : 3213 - 3223
  • [4] Burridge PW, 2014, NAT METHODS, V11, P855, DOI [10.1038/NMETH.2999, 10.1038/nmeth.2999]
  • [5] Infant with cardiomyopathy: When to suspect inborn errors of metabolism?
    Byers, Stephanie L.
    Ficicioglu, Can
    [J]. WORLD JOURNAL OF CARDIOLOGY, 2014, 6 (11): : 1149 - 1155
  • [6] Activation of aldehyde dehydrogenase-2 reduces ischemic damage to the heart
    Chen, Che-Hong
    Budas, Grant R.
    Churchill, Eric N.
    Disatnik, Marie-Helene
    Hurley, Thomas D.
    Mochly-Rosen, Daria
    [J]. SCIENCE, 2008, 321 (5895) : 1493 - 1495
  • [7] Reversal of Cardiac Dysfunction after Enzyme Replacement in Patients with Infantile-Onset Pompe Disease
    Chen, Lei-Ru
    Chen, Chun-An
    Chiu, Shuenn-Nan
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Lin, Ming-Tai
    Hwu, Wuh-Liang
    Wang, Jou-Kou
    Wu, Mei-Hwan
    [J]. JOURNAL OF PEDIATRICS, 2009, 155 (02) : 271 - 275
  • [8] A New Initiative on Precision Medicine
    Collins, Francis S.
    Varmus, Harold
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2015, 372 (09) : 793 - 795
  • [9] Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
    Ding, Qiurong
    Strong, Alanna
    Patel, Kevin M.
    Ng, Sze-Ling
    Gosis, Bridget S.
    Regan, Stephanie N.
    Cowan, Chad A.
    Rader, Daniel J.
    Musunuru, Kiran
    [J]. CIRCULATION RESEARCH, 2014, 115 (05) : 488 - +
  • [10] Disease Modeling and Phenotypic Drug Screening for Diabetic Cardiomyopathy using Human Induced Pluripotent Stem Cells
    Drawnel, Faye M.
    Boccardo, Stefano
    Prummer, Michael
    Delobel, Frederic
    Graff, Alexandra
    Weber, Michael
    Gerard, Regine
    Badi, Laura
    Kam-Thong, Tony
    Bu, Lei
    Jiang, Xin
    Hoflack, Jean-Christophe
    Kiialainen, Anna
    Jeworutzki, Elena
    Aoyama, Natsuyo
    Carlson, Coby
    Burcin, Mark
    Gromo, Gianni
    Boehringer, Markus
    Stahlberg, Henning
    Hall, Benjamin J.
    Magnone, Maria Chiara
    Kolaja, Kyle
    Chien, Kenneth R.
    Bailly, Jacques
    Iacone, Roberto
    [J]. CELL REPORTS, 2014, 9 (03): : 810 - 820