Single-AAV Delivery of the SpCas9 Nickase Improves CAG/CTG Repeat contraction in a mouse model of Huntington's disease

Single-AAV delivery of SpCas9 nickase with a shared promoter drives larger CAG/CTG repeat contractions in the striatum of a Huntington’s disease mouse model than a dual-vector system.

• Why it matters: Dual-vector AAV delivery is constrained by Cas9 size, limiting efficient in vivo genome editing for repeat contraction strategies in Huntington’s disease models.
• What they did: The authors packaged the full CRISPR system into one AAV using one promoter to express both SpCas9 nickase and a sgRNA, then compared single- versus dual-vector contraction in striatal tissue.
• The result: The all-in-one AAV system produced larger repeat contractions, supporting a more efficient, less costly route for in vivo gene editing and improving AAV translational potential.

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