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在生命早期高效的调制器治疗后,眼睛的发展

Yimin Zhu¹, Danni Li¹, Felisa Reyes-Ortega^1,2

¹Department of Biochemistry and Pharmacology, School of Biomedical Sciences, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Parkville, VIC, Australia.

Frontiers in pharmacology

|October 5, 2023

概括

高效的CFTR调节器疗法改善了CF患者的生活质量,导致更多的怀孕. 然而,这些疗法穿过胎盘进入母乳,可能会导致婴儿白内障的风险.

科学领域:

药理学药理学是指药理学的学科.
眼科医生眼科眼科
遗传学是一个遗传学.

背景情况:

高效的CFTR调制剂疗法 (HEMT) 已经改变了囊性纤维化治疗,改善了许多人的临床结果和生活质量.
由于健康状况改善,CF妇女的怀孕率增加,需要评估怀孕和哺乳期间的HEMT安全性.
已知HEMT穿过胎盘进入母乳,引发了对婴儿潜在毒性的担忧.

研究的目的:

审查有关HEMT对怀孕和母乳养期间透镜发育的影响现有证据.
探索HEMT可能影响正常和异常透镜发育的机制.
确定有关HEMT暴露和婴儿眼部健康的关键未回答的问题.

主要方法:

关于怀孕和哺乳期间HEMT暴露的临床前毒性研究和临床病例报告的文献综述.
对HEMT通过胎盘和进入母乳的传输数据的分析.
综合关于透镜发育和潜在的HEMT诱导白内障的信息.

主要成果:

在老鼠中进行的毒性研究表明,在暴露于HEMT后,婴儿白内障的风险存在.
病例报告表明,早年接触HEMT和先天性白内障之间存在相关性.
已证实,HEMT通过母体治疗转移到胎儿和婴儿.

结论:

怀孕和母乳养期间的HEMT暴露需要仔细考虑,因为婴儿白内障的潜在风险.
进一步的研究对于阐明HEMT对婴儿眼部发育的精确机制和长期影响至关重要.
临床监测和风险与益处的评估对于怀孕和哺乳期在HEMT上患有CF的患者至关重要.

关键词:

在CFTR调制器.卡塔拉拉特是什么?卡塔拉特是什么?囊性纤维化症是什么眼部形眼部形眼部形眼睛的发展眼睛的发展

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