Pluripotent stem cell-hereditary cardiomyopathy cardiac muscle cell and preparation method thereof

Abstract

The invention belongs to the field of researching and application of biomedicine and particularly relates to human pluripotent stem cell-hereditary cardiomyopathy cardiac muscle cells and a preparation method thereof. The invention provides a human hereditary cardiomyopathy-pluripotent stem cell, which is constructed by means of TALEN or CRISPR / CAS9 genome editing technology. The human hereditary cardiomyopathy cardiac muscle cell can be combined with any scaffold materials to culture various in-vitro human hereditary cardiomyopathy cardiac muscle tissues. The human hereditary cardiomyopathy cardiac muscle cells in the invention have disease phenotypes and electrophysiology change being similar as the cardiac muscle cells of human hereditary cardiomyopathy patients. The human hereditary cardiomyopathy cardiac muscle cells are wide in sources and can be cultured in-vitro for a long time. The invention provides excellent tools and platforms for researching an effective new therapy means and researching an effective corresponding treatment medicine.

Description

technical field [0001] The invention belongs to the field of biomedical research and application, and specifically relates to human pluripotent stem cells-hereditary cardiomyopathy cardiomyocytes and preparation technology thereof. Background technique [0002] Human hereditary cardiomyopathy includes five kinds of cardiomyopathy: hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), restricted cardiomyopathy, left ventricular noncompaction cardiomyopathy (left ventricular noncompaction, LVNC) and arrhythmogenic right ventricular cardiomyopathy (ARVC) are major heart diseases caused by multiple single gene mutations. These single genes include genes expressing sarcomere proteins, cytoskeletal proteins, ion channel proteins, lamins, mitochondrial proteins such as myosin heavy chain MHC, myosin light chain MLC, troponin TNNT2, myosin Binding protein MBPC3, sodium ion channel protein SCN5a, potassium ion channel protein KCNH2, nuclear lamin lamin, etc. (see Morita H...

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Problems solved by technology

[0005] It is very difficult to obtain the diseased human cardiomyocytes of inherited cardiomyopathy from the patient's heart, because the source is very rare and it is not easy to survive for a long time in vitro, and these diseased cardiomyocytes are already in the late stage of the disease, so there is not much research on the early mechanism of the disease. Multi-function

Although transgenic animal models of hereditary cardiomyopathy have also been established and have played an important role in the study of hereditary cardiomyopathy, animal models are quite different from humans due to the physiological function and molecular basis of the heart (such as the heart rate of mice). up to 500 times per second, which is 10 times that of human beings), and cannot fully reproduce the human disease phenotype, and the research on animal models of genetic cardiomyopathy is mostly concentrated in the late stage of the disease, and cannot be regarded as the cause of myocardial sarcomeres in the early stage of the disease. effective research object

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