Work

Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum

Year: 2025

Type: article

Abstract: defects through adenine base editing may represent a novel, permanent therapy for the treatment of PXE.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102769

Authors: Lauren C. Testa, Dora Obiri-Yeboah, Hooda Said, Ping Qu, Michael A. Levine, Mohamad-Gabriel Alameh, Kiran Musunuru, Qiaoli Li, Xiao Wang

Institutions: University of Pennsylvania, Thomas Jefferson University, Children's Hospital of Philadelphia, George Mason University

Language: English

Topic: Dermatological and Skeletal Disorders

Subfield: Genetics

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 Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institute of Child Health and Human Development, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Common Fund, NHLBI Division of Intramural Research

Awards: G1835707777, G2633039206, G7159601338, G7904603605

OpenAlex ID: W4416118337

FWCI:

Cites: 30

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