Cardiomyopathy in a Dish: Using Human Inducible Pluripotent Stem Cells to Model Inherited Cardiomyopathies

被引:13
作者
Kamdar, Forum
Kamdar, Andre Klaassen
Koyano-Nakagawa, Naoko
Garry, Mary G.
Garry, Daniel J. [1 ]
机构
[1] Univ Minnesota, Lillehei Heart Inst, Minneapolis, MN 55455 USA
基金
美国国家卫生研究院;
关键词
Cardiomyopathy; stem cells; disease models; LONG-QT SYNDROME; POLYMORPHIC VENTRICULAR-TACHYCARDIA; HYPERTROPHIC CARDIOMYOPATHY; DILATED CARDIOMYOPATHY; POMPE DISEASE; CARDIOMYOCYTES; DIFFERENTIATION; MECHANISMS; INDUCTION; MUTATION;
D O I
10.1016/j.cardfail.2015.04.010
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Inherited cardiomyopathies, including hypertrophic cardiomyopathy, dilated cardiomyopathies, arrythmogenic right ventricular cardiomyopathy, and other inherited forms of heart failure, represent a unique set of genetically defined cardiovascular disease processes. Unraveling the molecular mechanisms of these deadly forms of human heart disease has been challenging, but recent groundbreaking scientific advances in stem cell technology have allowed for the generation of patient-specific human inducible stem cell (hiPSC)-derived cardiomyocytes (CMs). hiPSC-derived CMs retain the genetic blueprint of the patient, they can be maintained in culture, and they recapitulate the phenotypic characteristics of the disease in vitro, thus serving as a disease in a dish. This review provides an overview of in vitro modeling of inherited cardiomyopathies with the use of patient-specific hiPSC-derived CMs.
引用
收藏
页码:761 / 770
页数:10
相关论文
共 65 条
[1]   Cardiac and Skeletal Muscle Defects in a Mouse Model of Human Barth Syndrome [J].
Acehan, Devrim ;
Vaz, Frederic ;
Houtkooper, Riekelt H. ;
James, Jeanne ;
Moore, Vicky ;
Tokunaga, Chonan ;
Kulik, Willem ;
Wansapura, Janaka ;
Toth, Matthew J. ;
Strauss, Arnold ;
Khuchua, Zaza .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2011, 286 (02) :899-908
[2]   Mechanisms of disease: molecular genetics of arrhythmogenic right ventricular dysplasia/cardiomyopathy [J].
Awad, Mark M. ;
Calkins, Hugh ;
Judge, Daniel P. .
NATURE CLINICAL PRACTICE CARDIOVASCULAR MEDICINE, 2008, 5 (05) :258-267
[3]   AN X-LINKED MITOCHONDRIAL DISEASE AFFECTING CARDIAC-MUSCLE, SKELETAL-MUSCLE AND NEUTROPHIL LEUKOCYTES [J].
BARTH, PG ;
SCHOLTE, HR ;
BERDEN, JA ;
VANDERKLEIVANMOORSEL, JM ;
LUYTHOUWEN, IEM ;
VANTVEERKORTHOF, ET ;
VANDERHARTEN, JJ ;
SOBOTKAPLOJHAR, MA .
JOURNAL OF THE NEUROLOGICAL SCIENCES, 1983, 62 (1-3) :327-355
[4]   Isogenic human pluripotent stem cell pairs reveal the role of a KCNH2 mutation in long-QT syndrome [J].
Bellin, Milena ;
Casini, Simona ;
Davis, Richard P. ;
D'Aniello, Cristina ;
Haas, Jessica ;
Ward-van Oostwaard, Dorien ;
Tertoolen, Leon G. J. ;
Jung, Christian B. ;
Elliott, David A. ;
Welling, Andrea ;
Laugwitz, Karl-Ludwig ;
Moretti, Alessandra ;
Mummery, Christine L. .
EMBO JOURNAL, 2013, 32 (24) :3161-3175
[5]   A novel X-linked gene, G4.5. is responsible for Barth syndrome [J].
Bione, S ;
DAdamo, P ;
Maestrini, E ;
Gedeon, AK ;
Bolhuis, PA ;
Toniolo, D .
NATURE GENETICS, 1996, 12 (04) :385-389
[6]   Production of De Novo Cardiomyocytes: Human Pluripotent Stem Cell Differentiation and Direct Reprogramming [J].
Burridge, Paul W. ;
Keller, Gordon ;
Gold, Joseph D. ;
Wu, Joseph C. .
CELL STEM CELL, 2012, 10 (01) :16-28
[7]   Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome [J].
Carvajal-Vergara, Xonia ;
Sevilla, Ana ;
D'Souza, Sunita L. ;
Ang, Yen-Sin ;
Schaniel, Christoph ;
Lee, Dung-Fang ;
Yang, Lei ;
Kaplan, Aaron D. ;
Adler, Eric D. ;
Rozov, Roye ;
Ge, YongChao ;
Cohen, Ninette ;
Edelmann, Lisa J. ;
Chang, Betty ;
Waghray, Avinash ;
Su, Jie ;
Pardo, Sherly ;
Lichtenbelt, Klaske D. ;
Tartaglia, Marco ;
Gelb, Bruce D. ;
Lemischka, Ihor R. .
NATURE, 2010, 465 (7299) :808-U12
[8]   Modeling of Arrhythmogenic Right Ventricular Cardiomyopathy With Human Induced Pluripotent Stem Cells [J].
Caspi, Oren ;
Huber, Irit ;
Gepstein, Amira ;
Arbel, Gil ;
Maizels, Leonid ;
Boulos, Monther ;
Gepstein, Lior .
CIRCULATION-CARDIOVASCULAR GENETICS, 2013, 6 (06) :557-568
[9]   Generation of Human Cardiomyocytes: A Differentiation Protocol from Feeder-free Human Induced Pluripotent Stem Cells [J].
Di Pasquale, Elisa ;
Song, Belle ;
Condorelli, Gianluigi .
JOVE-JOURNAL OF VISUALIZED EXPERIMENTS, 2013, (76)
[10]   New Therapeutic Approaches to Mendelian Disorders [J].
Dietz, Harry C. .
NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (09) :852-863