Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need

Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag

(2023) Shun-Qing Liang et al.   NATURE METHODS

Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing

(2023) Dominique L. Brooks et al.   Nature Communications

In vivo prime editing of a metabolic liver disease in mice

(2022) Desirée Böck et al.   Science Translational Medicine

The impact of metabolic control on cognition, neurophysiology, and well-being in PKU: A systematic review and meta-analysis of the within-participant literature

(2022) Lucie Thomas et al.   MOLECULAR GENETICS AND METABOLISM

TAPE-seq is a cell-based method for predicting genome-wide off-target effects of prime editor

(2022) Jeonghun Kwon et al.   Nature Communications

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enhanced prime editing systems by manipulating cellular determinants of editing outcomes

(2021) Peter J. Chen et al.   CELL

Engineered pegRNAs improve prime editing efficiency

(2021) James W. Nelson et al.   NATURE BIOTECHNOLOGY

The Genetic Landscape and Epidemiology of Phenylketonuria

(2020) Alicia Hillert et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up

(2020) Barbara K. Burton et al.   MOLECULAR GENETICS AND METABOLISM

CRISPResso2 provides accurate and rapid genome editing sequence analysis

(2019) Kendell Clement et al.   NATURE BIOTECHNOLOGY

Psychiatric and Cognitive Aspects of Phenylketonuria: The Limitations of Diet and Promise of New Treatments

(2019) Killian Ashe et al.   Frontiers in Psychiatry

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Adult cognitive outcomes in phenylketonuria: explaining causes of variability beyond average Phe levels

(2019) Cristina Romani et al.   Orphanet Journal of Rare Diseases

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

Adherence to clinic recommendations among patients with phenylketonuria in the United States

(2017) E.R. Jurecki et al.   MOLECULAR GENETICS AND METABOLISM

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Phenylalanine hydroxylase deficiency: diagnosis and management guideline

(2014)   GENETICS IN MEDICINE