Non-Invasive Detection of Allele-Specific CRISPR-SaCas9-KKH Disruption of TOR1A DYT1 Allele in a Xenograft Mouse Model

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The authors demonstrated a gene editing approach that selectively targeted the dominant 3-base pair deletion in the TOR1A DYT1 allele while safeguarding the wild-type TOR1A allele.
[Molecular Therapy – Nucleic Acids]
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