Allele-specific silencing of the gain-offunction mutation in huntington`s disease using crispr/cas9

HIGHLIGHTS

• who: Madelynn N. Whittaker and colleagues from the Center for GenomicPrinceton, New Jersey, USA have published the research work: Allele-specific silencing of the gain-offunction mutation in Huntington`s disease using CRISPR/Cas9, in the Journal: (JOURNAL) of August/26,/2022
• what: The authors developed a complementary CRISPR/Cas9 strategy that uses a single gRNA to selectively inactivate the mutant HTT through nonsense-mediated decay (NMD) , and the authors subsequently determined its applicability, allele-specificity, molecular consequences, and off-target effects to evaluate its utility in clinical applications. The authors reason that identification of . . .
  

   

  

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