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Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes.
Int J Mol Sci. 2022 Aug 16;23(16):9176. doi: 10.3390/ijms23169176.
Int J Mol Sci. 2022.
PMID: 36012442
Free PMC article.
Targeted-Deletion of a Tiny Sequence via Prime Editing to Restore SMN Expression.
Zhou M, Tang S, Duan N, Xie M, Li Z, Feng M, Wu L, Hu Z, Liang D.
Zhou M, et al.
Int J Mol Sci. 2022 Jul 19;23(14):7941. doi: 10.3390/ijms23147941.
Int J Mol Sci. 2022.
PMID: 35887289
Free PMC article.
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Cas14a1-Mediated Nucleic Acid Diagnostics for Spinal Muscular Atrophy.
Hu Z, Chen M, Zhang C, Li Z, Feng M, Wu L, Zhou M, Liang D.
Hu Z, et al.
Biosensors (Basel). 2022 Apr 23;12(5):268. doi: 10.3390/bios12050268.
Biosensors (Basel). 2022.
PMID: 35624569
Free PMC article.
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Ectopic Expression of FVIII in HPCs and MSCs Derived from hiPSCs with Site-Specific Integration of ITGA2B Promoter-Driven BDDF8 Gene in Hemophilia A.
Zhao J, Zhou M, Wang Z, Wu L, Hu Z, Liang D.
Zhao J, et al.
Int J Mol Sci. 2022 Jan 6;23(2):623. doi: 10.3390/ijms23020623.
Int J Mol Sci. 2022.
PMID: 35054807
Free PMC article.
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