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Items: 16


ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs.

Hu Z, Zhou M, Wu Y, Li Z, Liu X, Wu L, Liang D.

Mol Ther Nucleic Acids. 2019 Jun 5;17:198-209. doi: 10.1016/j.omtn.2019.05.019. [Epub ahead of print]


Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of F9 into rDNA Locus of Mouse ESCs.

Wang Y, Zhao J, Duan N, Liu W, Zhang Y, Zhou M, Hu Z, Feng M, Liu X, Wu L, Li Z, Liang D.

Int J Mol Sci. 2018 Oct 5;19(10). pii: E3035. doi: 10.3390/ijms19103035.


Ectopic expression of factor VIII in MSCs and hepatocytes derived from rDNA targeted hESCs.

Sun Q, Liu X, Wu Y, Niu W, Long P, Liu J, Lei M, Hu Y, Wu L, Li Z, Liang D.

Clin Chim Acta. 2019 Aug;495:656-663. doi: 10.1016/j.cca.2018.08.007. Epub 2018 Aug 7.


Generation of reporter hESCs by targeting EGFP at the CD144 locus to facilitate the endothelial differentiation.

Hu Z, Wu Y, Zhou M, Wang X, Pang J, Li Z, Feng M, Wang Y, Hu Q, Zhao J, Liu X, Wu L, Liang D.

Dev Growth Differ. 2018 May;60(4):205-215. doi: 10.1111/dgd.12433. Epub 2018 Apr 25.


Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells.

Zhou M, Hu Z, Qiu L, Zhou T, Feng M, Hu Q, Zeng B, Li Z, Sun Q, Wu Y, Liu X, Wu L, Liang D.

Hum Gene Ther. 2018 Nov;29(11):1252-1263. doi: 10.1089/hum.2017.255. Epub 2018 May 9.


Gene Therapy for Hemophilia and Duchenne Muscular Dystrophy in China.

Liu X, Liu M, Wu L, Liang D.

Hum Gene Ther. 2018 Feb;29(2):146-150. doi: 10.1089/hum.2017.213. Review.


Restoration of SMN expression in mesenchymal stem cells derived from gene-targeted patient-specific iPSCs.

Feng M, Liu C, Xia Y, Liu B, Zhou M, Li Z, Sun Q, Hu Z, Wang Y, Wu L, Liu X, Liang D.

J Mol Histol. 2018 Feb;49(1):27-37. doi: 10.1007/s10735-017-9744-1. Epub 2017 Dec 5.


Enhanced tumor growth inhibition by mesenchymal stem cells derived from iPSCs with targeted integration of interleukin24 into rDNA loci.

Liu B, Chen F, Wu Y, Wang X, Feng M, Li Z, Zhou M, Wang Y, Wu L, Liu X, Liang D.

Oncotarget. 2017 Jun 20;8(25):40791-40803. doi: 10.18632/oncotarget.16584.


Targeting of the human F8 at the multicopy rDNA locus in Hemophilia A patient-derived iPSCs using TALENickases.

Pang J, Wu Y, Li Z, Hu Z, Wang X, Hu X, Wang X, Liu X, Zhou M, Liu B, Wang Y, Feng M, Liang D.

Biochem Biophys Res Commun. 2016 Mar 25;472(1):144-9. doi: 10.1016/j.bbrc.2016.02.083. Epub 2016 Feb 24.


Pathogenic Ubqln2 gains toxic properties to induce neuron death.

Wu Q, Liu M, Huang C, Liu X, Huang B, Li N, Zhou H, Xia XG.

Acta Neuropathol. 2015 Mar;129(3):417-28. doi: 10.1007/s00401-014-1367-y. Epub 2014 Nov 12.


Pathogenic mutation of UBQLN2 impairs its interaction with UBXD8 and disrupts endoplasmic reticulum-associated protein degradation.

Xia Y, Yan LH, Huang B, Liu M, Liu X, Huang C.

J Neurochem. 2014 Apr;129(1):99-106. doi: 10.1111/jnc.12606. Epub 2013 Nov 22.


Nonviral gene targeting at rDNA locus of human mesenchymal stem cells.

Hu Y, Liu X, Long P, Xiao D, Cun J, Li Z, Xue J, Wu Y, Luo S, Wu L, Liang D.

Biomed Res Int. 2013;2013:135189. doi: 10.1155/2013/135189. Epub 2013 May 14.


Expression of ALS-linked TDP-43 mutant in astrocytes causes non-cell-autonomous motor neuron death in rats.

Tong J, Huang C, Bi F, Wu Q, Huang B, Liu X, Li F, Zhou H, Xia XG.

EMBO J. 2013 Jul 3;32(13):1917-26. doi: 10.1038/emboj.2013.122. Epub 2013 May 28.


Targeting of the human coagulation factor IX gene at rDNA locus of human embryonic stem cells.

Liu X, Wu Y, Li Z, Yang J, Xue J, Hu Y, Feng M, Niu W, Yang Q, Lei M, Xia J, Wu L, Liang D.

PLoS One. 2012;7(5):e37071. doi: 10.1371/journal.pone.0037071. Epub 2012 May 16.


A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus.

Yang J, Liu X, Yu J, Sheng L, Shi Y, Li Z, Hu Y, Xue J, Wu L, Liang Y, Xia J, Liang D.

Acta Biochim Biophys Sin (Shanghai). 2009 Dec;41(12):1053-60.


Tissue specific expression of suicide genes delivered by nanoparticles inhibits gastric carcinoma growth.

Liu T, Zhang G, Chen YH, Chen Y, Liu X, Peng J, Xu MH, Yuan JW.

Cancer Biol Ther. 2006 Dec;5(12):1683-90. Epub 2006 Dec 20.


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