Article

Treatment of a metabolic liver disease by <i>in vivo</i> prime editing in mice

2021-08-17

Abstract excerpt

Prime editing is a highly versatile CRISPR-based genome editing technology with the potential to correct the vast majority of pathogenic mutations (1) . However, correction of a disease phenotype in vivo in somatic tissues has not been demonstrated thus far. Here, we establish proof-of-concept for in vivo prime editing and repair the metabolic liver disease phenylketonuria (PKU) in mice. We first developed a si...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 1ce589f6-448b-5e64-bedf-ab9dd42d4d5d
DOI: 10.1101/2021.08.17.456632

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Treatment of a metabolic liver disease by <i>in vivo</i> prime editing in miceDOI 10.1101/2021.08.17.456632Read

In vivo prime editing of a metabolic liver disease in miceSemantic proximity Read

Treatment of a genetic liver disease in mice through transient prime editor expressionSemantic proximity Read

Treatment of a metabolic liver disease in mice with a transient prime editing approachSemantic proximity Read

Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases.Semantic proximity Read

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice.Semantic proximity Read

Improved prime editors enable pathogenic allele correction and cancer modelling in adult miceSemantic proximity Read

Prime Editing for Human Gene Therapy: Where Are We Now?Semantic proximity Read

Prime editing enables precise genome editing in mouse liver and retinaSemantic proximity Read

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn miceSemantic proximity Read

Optimizing twin prime editing components for scalable genome editing and therapy in spinocerebellar ataxia type 3Semantic proximity Read

Efficient prime editing in mouse brain, liver and heart with dual AAVsSemantic proximity Read

Improved specificity and efficiency of <i>in vivo</i> adenine base editing therapies with hybrid guide RNAsSemantic proximity Read