Ocular development after highly effective modulator treatment early in life

Frontiers in Pharmacology

Pharmacology

Yimin Zhu¹  Danni Li¹  Elena K. Schneider-Futschik¹  Felisa Reyes-Ortega²  Holly R. Chinnery³ 

[1] Department of Biochemistry and Pharmacology, School of Biomedical Sciences, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Parkville, VIC, Australia;Department of Biochemistry and Pharmacology, School of Biomedical Sciences, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Parkville, VIC, Australia;Department of Ophthalmology, Maimonides Biomedical Research Institute of Cordoba (IMIBIC), Reina Sofia University Hospital and University of Cordoba, Cordoba, Spain;Department of Optometry and Vision Sciences, The University of Melbourne, Parkville, VIC, Australia;

关键词: cystic fibrosis;    ocular development;    CFTR modulator;    catarat;    eye malformations;   

DOI  :  10.3389/fphar.2023.1265138

 received in 2023-07-22, accepted in 2023-09-06,  发布年份 2023

来源: Frontiers

【 摘 要 】

Highly effective cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator therapies (HEMT), including elexacaftor-tezacaftor-ivacaftor, correct the underlying molecular defect causing CF. HEMT decreases general symptom burden by improving clinical metrics and quality of life for most people with CF (PwCF) with eligible CFTR variants. This has resulted in more pregnancies in women living with CF. All HEMT are known to be able pass through the placenta and into breast milk in mothers who continue on this therapy while pregnant and breast feeding. Toxicity studies of HEMT in young rats demonstrated infant cataracts, and case reports have reported the presence of congenital cataracts in early life exposure to HEMT. This article reviews the evidence for how HEMT influences the dynamic and interdependent processes of healthy and abnormal lens development in the context of HEMT exposure during pregnancy and breastfeeding, and raises questions that remain unanswered.

【 授权许可】

Unknown   
Copyright © 2023 Zhu, Li, Reyes-Ortega, Chinnery and Schneider-Futschik.