| Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific … M Zhou, Z Hu, L Qiu, T Zhou, M Feng, Q Hu, B Zeng, Z Li, Q Sun, Y Wu, ... Human gene therapy 29 (11), 1252-1263, 2018 | 62 | 2018 |
| Modeling Alzheimer’s disease with induced pluripotent stem cells: current challenges and future concerns W Zhang, B Jiao, M Zhou, T Zhou, L Shen Stem Cells International 2016, 2016 | 34 | 2016 |
| Targeting of the human F8 at the multicopy rDNA locus in hemophilia A patient-derived iPSCs using TALENickases J Pang, Y Wu, Z Li, Z Hu, X Wang, X Hu, X Wang, X Liu, M Zhou, B Liu, ... Biochemical and biophysical research communications 472 (1), 144-149, 2016 | 33 | 2016 |
| ssODN-mediated in-frame deletion with CRISPR/Cas9 restores FVIII function in hemophilia A-patient-derived iPSCs and ECs Z Hu, M Zhou, Y Wu, Z Li, X Liu, L Wu, D Liang Molecular Therapy-Nucleic Acids 17, 198-209, 2019 | 29 | 2019 |
| Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of F9 into rDNA Locus of Mouse ESCs Y Wang, J Zhao, N Duan, W Liu, Y Zhang, M Zhou, Z Hu, M Feng, X Liu, ... International Journal of Molecular Sciences 19 (10), 3035, 2018 | 26 | 2018 |
| Enhanced tumor growth inhibition by mesenchymal stem cells derived from iPSCs with targeted integration of interleukin24 into rDNA loci B Liu, F Chen, Y Wu, X Wang, M Feng, Z Li, M Zhou, Y Wang, L Wu, X Liu, ... Oncotarget 8 (25), 40791, 2017 | 25 | 2017 |
| Targeted-deletion of a tiny sequence via prime editing to restore SMN expression M Zhou, S Tang, N Duan, M Xie, Z Li, M Feng, L Wu, Z Hu, D Liang International Journal of Molecular Sciences 23 (14), 7941, 2022 | 20 | 2022 |
| Site-Specific Integration of TRAIL in iPSC-Derived Mesenchymal Stem Cells for Targeted Cancer Therapy Z Wang, H Chen, P Wang, M Zhou, G Li, Z Hu, Q Hu, J Zhao, X Liu, L Wu, ... Stem Cells Translational Medicine 11 (3), 297-309, 2022 | 15 | 2022 |
| Amyotrophic lateral sclerosis (ALS) linked mutation in Ubiquilin 2 affects stress granule assembly via TIA‐1 G Peng, A Gu, H Niu, L Chen, Y Chen, M Zhou, Y Zhang, J Liu, L Cai, ... CNS Neuroscience & Therapeutics 28 (1), 105-115, 2022 | 13 | 2022 |
| CRISPR/Cas12a-Based Ultrasensitive and Rapid Detection of JAK2 V617F Somatic Mutation in Myeloproliferative Neoplasms M Chen, C Zhang, Z Hu, Z Li, M Li, L Wu, M Zhou, D Liang Biosensors 11 (8), 247, 2021 | 10 | 2021 |
| Cas12a and lateral flow strip-based test for rapid and ultrasensitive detection of spinal muscular atrophy C Zhang, Z Li, M Chen, Z Hu, L Wu, M Zhou, D Liang Biosensors 11 (5), 154, 2021 | 10 | 2021 |
| Loss of PIGK function causes severe infantile encephalopathy and extensive neuronal apoptosis X Chen, W Yin, S Chen, W Zhang, H Li, H Kuang, M Zhou, Y Teng, ... Human Genetics 140, 791-803, 2021 | 9 | 2021 |
| Targeted addition of mini-dystrophin into rDNA locus of Duchenne muscular dystrophy patient-derived iPSCs B Zeng, M Zhou, B Liu, F Shen, R Xiao, J Su, Z Hu, Y Zhang, A Gu, L Wu, ... Biochemical and Biophysical Research Communications 545, 40-45, 2021 | 8 | 2021 |
| Restoration of FVIII Function and Phenotypic Rescue in Hemophilia A Mice by Transplantation of MSCs Derived From F8-Modified iPSCs L Qiu, M Xie, M Zhou, X Liu, Z Hu, L Wu Frontiers in Cell and Developmental Biology 9, 630353, 2021 | 8 | 2021 |
| Gene therapy for hemophilia A: where we stand M Zhou, Z Hu, C Zhang, L Wu, Z Li, D Liang Current Gene Therapy 20 (2), 142-151, 2020 | 7 | 2020 |
| Restoration of SMN expression in mesenchymal stem cells derived from gene-targeted patient-specific iPSCs M Feng, C Liu, Y Xia, B Liu, M Zhou, Z Li, Q Sun, Z Hu, Y Wang, L Wu, ... Journal of Molecular Histology 49, 27-37, 2018 | 7 | 2018 |
| Targeted B-domain deletion restores F8 function in human endothelial cells and mice Z Hu, Z Li, Y Wu, J Zhao, L Wu, M Zhou, D Liang Signal Transduction and Targeted Therapy 7 (1), 189, 2022 | 6 | 2022 |
| IL-24 armored CAR19-T cells show enhanced antitumor activity and persistence Q Hu, Y Zhang, P Wang, M Zhou, Z Hu, C Liu, M Liu, L Wu, X Liu, D Liang Signal Transduction and Targeted Therapy 6 (1), 14, 2021 | 6 | 2021 |
| Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes R Xiao, M Zhou, P Wang, B Zeng, L Wu, Z Hu, D Liang International Journal of Molecular Sciences 23 (16), 9176, 2022 | 5 | 2022 |
| Ectopic Expression of FVIII in HPCs and MSCs Derived from hiPSCs with Site-Specific Integration of ITGA2B Promoter-Driven BDDF8 Gene in Hemophilia A J Zhao, M Zhou, Z Wang, L Wu, Z Hu, D Liang International Journal of Molecular Sciences 23 (2), 623, 2022 | 5 | 2022 |
