Work

Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants

Year: 2025

Type: article

Abstract: correction of the second and third most common pathogenic variants of PKU.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102770

Authors: Aidan Quigley, Ishaan Jindal, Thomas Campion, Delaney Rutherford, Yongseok Han, Walsh Quigley, Ping Qu, Kiran Musunuru, Rebecca Ahrens-Nicklas, Xinying Hong, Xiao Wang

Institutions: University of Pennsylvania, Children's Hospital of Philadelphia

Language: English

Topic: CRISPR and Genetic Engineering

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900

Funders: National Institutes of Health, National Heart, Lung, and Blood Institute, National Institute of Child Health and Human Development, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Common Fund

Awards: G2633039206, G4095291439, G7159601338

OpenAlex ID: W4416118495

FWCI: 1.044

Cites: 19

Cited by: 4

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