Improvement of cardiac function by short-term enzyme replacement therapy in a murine model of cardiomyopathy associated with Hunter syndrome evaluated by serial echocardiography with speckle tracking 2-D strain analysis.
Cardiac systolic function is significantly decreased in a proportion of patients with Hunter syndrome. This study was performed to evaluate the change in myocardial function associated with enzyme replacement therapy (ERT) in a mouse model of cardiomyopathy associated with Hunter syndrome. Thirty 9-week-old iduronate-2-sulfatase (IDS) knockout mice received either intravenous injection of human recombinant IDS (ERT group, N=15) or saline (control group, N=15) for 5 weeks. Echocardiography was performed at baseline and after treatment. Echocardiographic parameters of left ventricular (LV) systolic function and 2-dimensional radial and circumferential strain were assessed. At follow-up, there was a significant increase in LV fractional shortening and radial and circumferential strain in the ERT group only. Notable myocardial fibrosis was observed in the control group only. In the murine model of Hunter syndrome, ERT exerts beneficial effects on cardiac function, which can be evaluated by serial echocardiographic evaluation including 2-dimensional strain analysis.
Heart, Vascular and Stroke Institute, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Sang-Chol
SC
Lee
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Jieun
J
Lee
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea. Electronic address: jindk@skku.edu.
Dong-Kyu
DK
Jin
Department of Pathology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Jung-Sun
JS
Kim
Heart, Vascular and Stroke Institute, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Eun-Seok
ES
Jeon
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Young Hee
YH
Kwun
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Mi Sun
MS
Chang
Clinical Research Center, Samsung Biomedical Research Center, Seoul, Republic of Korea.
Ah-ra
AR
Ko
Clinical Research Center, Samsung Biomedical Research Center, Seoul, Republic of Korea.
Yeon Joo
YJ
Yook
Department of Medical Genetics, Ajou University School of Medicine, Suwon, Republic of Korea.
Young Bae
YB
Sohn
D000818:Animals; D009202:Cardiomyopathies / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology* / Q000503:physiopathology; D004195:Disease Models, Animal; D004452:Echocardiography; D056947:Enzyme Replacement Therapy; D005260:Female; D007066:Iduronate Sulfatase / Q000627:therapeutic use*; D008297:Male; D051379:Mice; D018345:Mice, Knockout; D016532:Mucopolysaccharidosis II / Q000150:complications* / Q000188:drug therapy*; D016896:Treatment Outcome; D018487:Ventricular Dysfunction, Left / Q000188:drug therapy
D007066:Iduronate Sulfatase
Echocardiography; Enzyme replacement; Mucopolysaccharidosis; Speckle tracking; Strain