Work

Novel treatment for PXE: Recombinant ENPP1 enzyme therapy

Year: 2024

Type: article

Abstract: mice were injected weekly from 5 to 15 weeks of age, BL-1118 dose-dependently increased steady-state plasma ENPP1 activity and PPi levels and significantly reduced ectopic calcification in the muzzle skin and kidneys. These results suggest that BL-1118 is a promising second generation enzyme therapy for PXE, the first generation of which is currently in clinical testing.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2024.09.028

Authors: Ida Joely Jacobs, Dora Obiri-Yeboah, Paul R. Stabach, Demetrios T. Braddock, Qiaoli Li

Institutions: Thomas Jefferson University, Yale 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: hybrid

Estimated APC paid: $3,900