CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
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Cites methods from "CRISPR-Cpf1 correction of muscular ..."
...Immunostaining iPSC-derived cardiomyocytes were fixed with acetone and subjected to immunostaining, as described previously (25)....
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...Cas9 and guide RNAs for correction or bypass of the mutations in iPSC lines were introduced into cells by nucleofection, as described previously (25)....
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...Western blot analysis Western blot analysis for human iPSC–derived cardiomyocytes was performed, as described previously (25), using antibodies to dystrophin (ab15277, Abcam; D8168, Sigma-Aldrich), glyceraldehyde-3-phosphate dehydrogenase (MAB374, Millipore), and cardiac myosin heavy chain (ab50967, Abcam)....
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...Pools of treated cells or single clones were then differentiated into induced cardiomyocytes (iCMs) using standardized conditions (25, 32)....
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186 citations
Cites background from "CRISPR-Cpf1 correction of muscular ..."
...In addition, we have shown that the Cas9-related endonuclease Cpf1 can correct the Dmd mutation in mdx mice (20)....
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References
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"CRISPR-Cpf1 correction of muscular ..." refers methods in this paper
...Immunohistochemistry of skeletal muscles, heart, and brain was performed, as previously described (7, 12), using a dystrophin antibody (MANDYS8, 1:800; Sigma-Aldrich)....
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...net/ cas-offinder/), and off-target analysis was performed as previously described (12)....
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"CRISPR-Cpf1 correction of muscular ..." refers background in this paper
...The CRISPR (clustered regularly interspaced short palindromic repeats) system functions as an adaptive immune system in bacteria and archaea that defends against phage infection (5)....
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