Decoding the complexity of on-target integration: characterizing DNA insertions at the CRISPR-Cas9 targeted locus using nanopore sequencing

Decoding the complexity of on-target integration: characterizing DNA insertions at the CRISPR-Cas9 targeted locus using nanopore sequencing

Abstract Background CRISPR-Cas9 technology has advanced in vivo gene therapy for disorders like hemophilia A, notably through the successful targeted incorporation of the F8 gene into the Alb locus in hepatocytes, effectively curing this disorder in mice. However, thoroughly evaluating the safety an...

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Bibliographic Details
Main Authors: Juan-Juan Zhao, Xin-Yu Sun, Sai-Ning Tian, Zong-Ze Zhao, Meng-Di Yin, Mei Zhao, Feng Zhang, Si-Ang Li, Zhi-Xue Yang, Wei Wen, Tao Cheng, An Gong, Jian-Ping Zhang, Xiao-Bing Zhang
Format: Article
Language:English
Published: BMC 2024-02-01
Series:BMC Genomics
Subjects:
CRISPR-Cas9
Gene therapy
DNA integration
Long-range PCR
Nanopore sequencing
Online Access:https://doi.org/10.1186/s12864-024-10050-6

Internet

https://doi.org/10.1186/s12864-024-10050-6

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