Efficient Correction of a Hypertrophic Cardiomyopathy Mutation by ABEmax-NG

Shuhong Ma; Wenjian Jiang; Xujie Liu; Wen-Jing Lu; Tao Qi; Jingjing Wei; Fujian Wu; Yun Chang; Siyao Zhang; Yabing Song; Rui Bai; Jianbing Wang; Andrew S Lee; Hongjia Zhang; Yongming Wang; Feng Lan

doi:10.1161/CIRCRESAHA.120.318674

Efficient Correction of a Hypertrophic Cardiomyopathy Mutation by ABEmax-NG

Circ Res. 2021 Oct 29;129(10):895-908. doi: 10.1161/CIRCRESAHA.120.318674. Epub 2021 Sep 16.

Authors

Shuhong Ma^#^{1

2

3

4}, Wenjian Jiang^#^{3

4}, Xujie Liu^#⁵, Wen-Jing Lu^{1

2}, Tao Qi⁶, Jingjing Wei^{7

6}, Fujian Wu^{3

4}, Yun Chang^{3

4}, Siyao Zhang^{3

4}, Yabing Song⁷, Rui Bai^{3

4}, Jianbing Wang, Andrew S Lee^{8

9}, Hongjia Zhang^{3

4}, Yongming Wang⁶, Feng Lan^{2

3

4

5}

Affiliations

¹ Fuwai Hospital Chinese Academy of Medical Sciences, Shenzhen, State Key Laboratory of Cardiovascular Disease, Shenzhen (S.M., W.-J.L.).
² State Key Laboratory of Cardiovascular Disease, National Center for Cardiovascular Diseases, Fuwai Hospital, Key Laboratory of Application of Pluripotent Stem Cells in Heart Regeneration, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing (S.M., W.-J.L., F.L.).
³ Beijing Laboratory for Cardiovascular Precision Medicine, The Key Laboratory of Biomedical Engineering for Cardiovascular Disease Research, The Key Laboratory of Remodeling-Related Cardiovascular Disease, Ministry of Education, Beijing Anzhen Hospital, Capital Medical University (S.M., W.J., F.W., Y.C., S.Z., R.B., H.Z., F.L.).
⁴ Beijing Institute of Heart, Lung and Blood Vessel Diseases (S.M., W.J., F.W., Y.C., S.Z., R.B., H.Z., F.L.).
⁵ Shenzhen Key Laboratory of Cardiovascular Disease, Fuwai Hospital Chinese Academy of Medical Sciences (X.L., F.L.).
⁶ State Key Laboratory of Genetic Engineering, School of Life Sciences, Zhongshan Hospital, Fudan University (T.Q., J.W., Y.W.).
⁷ School of Life Sciences, and Tsinghua-Peking Center for Life Sciences, Tsinghua University (Y.S., J.W.).
⁸ Institute for Cancer Research, Shenzhen Bay Laboratory (A.S.L.).
⁹ Peking University Shenzhen Graduate School (A.S.L.).

^# Contributed equally.

PMID: 34525843
DOI: 10.1161/CIRCRESAHA.120.318674

Abstract

[Figure: see text].

Keywords: base editor; gene therapy; genome editing; hypertrophic cardiomyopathy.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Animals
Cardiomyopathy, Hypertrophic / genetics
Cardiomyopathy, Hypertrophic / therapy*
Cells, Cultured
Gene Editing / methods
Genetic Therapy / methods*
HEK293 Cells
Humans
Male
Mice
Mice, Inbred C57BL
Mutation
Myosin Heavy Chains / genetics*
Myosin Heavy Chains / metabolism

Substances

Myh6 protein, mouse
Myosin Heavy Chains