Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice

HIGHLIGHTS

• who: Daniel Reichart from the (UNIVERSITY) have published the research: Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice, in the Journal: (JOURNAL) of 16/Dec/2022
• what: The authors assessed two different genetic therapies-an adenine base editor (ABE8e) and a potent Cas9 nuclease delivered by AAV9-to prevent disease in mice carrying the heterozygous HCM pathogenic variant myosin R403Q. The authors report genomic editing corrected over 70% of mutant transcripts, with tolerable frequencies of edited bystander nucleotides (for example, proximal to the targeted adenosine residue). The authors aimed to selectively inactivate the . . .
  

   

  

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