主要开展遗传性心脏病研究,建立了首个肥厚型心肌病 iPS 模型(Cell Stem Cell 2013#, Web of SCI 引用>500)。带领团队采用碱基编辑器(Base Editor)最早实现了肥厚型心肌病模型的胚胎期基因编辑修正治疗(Circulation Research,2021*),和成体遗传性心律失常的转录水平的完全修正(Circulation,2024*),并基于该研究获批美国FDA治疗LQT3的孤儿药认定。以通讯作者在European Heart Journal (2025*),Circulation (2024*)、Nature Biomedical Engineering (2023*)、Science Advances (2023*)、Circulation Research (2021*)、Cell Discovery (2024*)、Bioactive Material (2021*)、Nature Communication (2019*, 2018*),Cell Death Differentiation (2019*)等高水平杂志发表论文50篇。 获批国际专利一项,中国发明专利6项。
代表性成果
1.Qi M#, Ma S# Liu J#, Liu X, Wei J, Lu W, Zhang S, Chang Y, Zhang Y, Zhong K, Yan Y, Zhu, M, Song Y, Chen Y, Hao G, Wang J, Wang L, Lee A, Chen X, Wang Y*, Lan F*. In vivo base editing of Scn5a rescues type 3 long QT syndrome in mice. Circulation. 2024, 149(4): 317-332. (IF=37.8)
2.Wang Y, Qi T, Liu J, Yang Y, Wang Z, Wang Y, Wang T, Li M, Li M, Lu D, Chang ACY, Yang L, Gao S, Wang Y, Lan F*. A highly specific CRISPR-Cas12j nuclease enables allele-specific genome editing. Sci Adv. 2023 Feb 10;9(6).(IF=13.6)
3.You Y, Tian Y, Yang Z, Shi J, Kwak KJ, Tong Y, Estania AP, Cao J, Hsu WH, Liu Y, Chiang CL, Schrank BR, Huntoon K, Lee D, Li Z, Zhao Y, Zhang H, Gallup TD, Ha J, Dong S, Li X, Wang Y, Lu WJ, Bahrani E, Lee LJ, Teng L, Jiang W, Lan F*, Kim BYS*, Lee AS*. Intradermally delivered mRNA-encapsulating extracellular vesicles for collagen-replacement therapy. Nat Biomed Eng. 2023 Jul;7(7):887-900 (IF=28.1)
4.Zhou P, Feng Fa, Song Y, Lia J, Li Q, Xu Z, Shi J, Qin L, He F, Li H, Han Y,Zhang R, Liu H*, Lan F*. Novel RGD-containing peptides exhibited improved abilities to integrin receptor binding and cultures of human induced pluripotent stem cells. Materials & Design. 2022 July 219:110762. (IF=9.4167)
5.Ma S, Jiang W, Liu X, Lu W, Qi t, Wei J, Wu F, Chang Y, Zhang S, Song Y, Bai R, Wang J, Lee A, Zhang H*, Wang Y*, Lan F*. Efficient correction of a hypertrophic cardiomyopathy mutation by ABEmax-NG. Circulation Research, 2021, Oct 29;129(10):895-908. (IF=20.1)
6.Guo R; Wan F; Xu Q; Feng T; Yang H; Gong Y; Ma S; Chang Y; Zhang S; Jiang Y, Wang H; Chang D; Zhang H; Ling Y*; Lan F*. Cell sheet formation enhances the therapeutic effects of human umbilical cord mesenchymal stem cells on myocardial infarction as a bioactive material.BioactiveMaterial, 2021, Mar 5;6(9):2999-3012.(IF=18.9)
7.Jiang Y, Li X, Guo T, Lu WJ, Ma S, Chang Y, Song Y, Zhang S, Bai R, Wang H, Qi M, Jiang H*, Zhang H*, Lan F*. Ranolazine rescues the heart failure phenotype of PLN-deficient human pluripotent stem cell-derived cardiomyocytes. Stem Cell Reports. 2022 Apr 12;17(4):804-819. (IF=5.9)
8.Wang D, Zhang C, Wang B, Li B, Wang Q, Liu D, Wang H, Zhou Y, Shi L,Lan F*, Wang Y*.Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning. Nature Communication. 2019 Sep 19;10(1):4284.(IF=16.6)
9.Lan F#, Lee AS#, Liang P#, Sanchez-Freire V, Nguyen PK , Wang L, Han L, Yen M, Wang Y, Sun N, Abilez OJ, Navarrete EG, Wheeler M, Lewis R, Yamaguchi Y, Ashley EA, Bers DM, Robbins RC, Longaker MT, Wu JC. Abnormal Calcium Handling Properties Underlie Familial Hypertrophic Cardiomyopathy Pathology in Patient-Specific Induced Pluripotent Stem Cells. CellStem Cell. 2013;12:101-113.(IF=23.9)
10.Lan F#, Liu J#, Narsinh KH, Hu S, Han L, Lee AS, Karow M, Nguyen PK, Nag D, Calos MP, Robbins RC, Wu JC*. Safe genetic modification of cardiac stem cells using a site-specific integration technique. Circulation. 2012;126:S20-28(IF=37.8)