What question did this study set out to answer?

Evaluate whether adenine base editing and prime editing can correct dystrophin gene mutations and rescue protein expression in cell and animal models of Duchenne muscular dystrophy.

April 30, 2021Science AdvancesOpen Access

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

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Authors

FCFrancesco ChemelloThe University of Texas Southwestern Medical Center ACAndreas C. ChaiThe University of Texas Southwestern Medical Center HLHui LiNanjing Agricultural University

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Implication

Preclinical study demonstrates targeted base and prime editing restore dystrophin in cellular and mouse models of muscular dystrophy, highlighting a precise therapeutic gene-editing strategy.

Key Points

Evaluate whether adenine base editing and prime editing can correct dystrophin gene mutations and rescue protein expression in cell and animal models of Duchenne muscular dystrophy.
Targeted splice donor sites with an adenine base editor to induce exon 51 skipping and used prime editing to restore the dystrophin reading frame in human induced pluripotent stem cell-derived cardiomyocytes.
Delivered adenine base editor components as a split-intein trans-splicing system via intramuscular injection of adeno-associated virus serotype 9 into exon 51-deleted mice.
Both adenine base editing and prime editing successfully restored dystrophin protein expression in human cardiomyocytes carrying exon 51 deletions.
Viral delivery of split-intein adenine base editor machinery achieved targeted in vivo nucleotide editing and functional disease correction in dystrophic mice.

Cite This Study

Chemello et al. (2021) studied this question.

synapsesocial.com/papers/69da26050f32475823a3d249 https://doi.org/10.1126/sciadv.abg4910

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Implication

Preclinical study demonstrates targeted base and prime editing restore dystrophin in cellular and mouse models of muscular dystrophy, highlighting a precise therapeutic gene-editing strategy.

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

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Implication

Key Points

Cite This Study

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

Discussion

Member takes

Implication

Key Points

Cite This Study

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