Lentiviral Gene Transfer to iPS Cells: Toward the Cardiomyocyte Differentiation of Pompe Disease-Specific iPS Cells

Yohei Sato; Takashi Higuchi; Hiroshi Kobayashi; Susumu Minamisawa; Hiroyuki Ida; Toya Ohashi

doi:10.1007/978-4-431-54628-3_48

Lentiviral Gene Transfer to iPS Cells: Toward the Cardiomyocyte Differentiation of Pompe Disease-Specific iPS Cells

Review

In: Etiology and Morphogenesis of Congenital Heart Disease: From Gene Function and Cellular Interaction to Morphology [Internet]. Tokyo: Springer; 2016. Chapter 48.

2016 Jun 25.

Authors

Yohei Sato^{1

2}, Takashi Higuchi², Hiroshi Kobayashi^{1

2}, Susumu Minamisawa³, Hiroyuki Ida^{1

2}, Toya Ohashi^{1

2}

Book Editors

Toshio Nakanishi¹, Roger R. Markwald², H.Scott Baldwin³, Bradley B. Keller⁴, Deepak Srivastava⁵, Hiroyuki Yamagishi⁶

Affiliations

¹ Department of Pediatrics, Jikei University School of Medicine, 3-25-8 Nishishimbashi Minato-ku, Tokyo, Japan
² Department of Gene Therapy, Jikei University School of Medicine, 3-25-8 Nishishimbashi Minato-ku, Tokyo, Japan
³ Department of Cell Physiology, Jikei University School of Medicine, 3-25-8 Nishishimbashi Minato-ku, Tokyo, Japan

Book Affiliations

¹ Department of Pediatric Cardiology, Tokyo Women's Medical University, Tokyo, Japan
² Cardiovascular Developmental Biology Center, Medical University of South Carolina Clemson University, Charleston, South Carolina, USA
³ Department of Pediatrics (Cardiology), Vanderbilt University, Nashville, Tennessee, USA
⁴ Pediatrics, Pharmacology and Toxicology, and Bioengineering, University of Louisville, Louisville, Kentucky, USA
⁵ Gladstone Institute of Cardiovascular Disease, San Francisco, California, USA
⁶ Pediatrics, Division of Pediatric Cardiology, Keio University School of Medicine, Tokyo, Japan

Excerpt

Pompe disease is an inherited neuromuscular disorder caused by a genetic deficiency of acid-glucosidase-alpha (GAA). The clinical symptoms of Pompe disease include progressive weakness, respiratory failure, and ventricular hypertrophy. Enzyme replacement therapy has been shown to ameliorate these symptoms. Cardiomyocytes derived from patient/disease-specific iPS cells (iPS-CMs) have been used for pathophysiological analyses, drug screening, and cell therapy. Our research goal was to generate cardiomyocytes that can be differentiated from gene-corrected Pompe disease-specific iPS cells.

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