Current Status and Prospects of Gene Therapy for Primary Ciliary Dyskinesia

Primary ciliary dyskinesia (PCD) is a genetically heterogeneous disease characterized by structural or functional abnormalities of motile cilia. It often presents clinically with recurrent respiratory infections, situs inversus, hydrocephalus, and infertility. Currently, there is no clinical treatme...

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Bibliographic Details
Published in:罕见病研究
Main Authors: LU Wanqing, LI Yixuan, HE Miao, TIAN Xinlun, LIU Yaping
Format: Article
Language:Chinese
Published: Editorial Office of Journal of Rare Diseases 2025-07-01
Subjects:
primary ciliary dyskinesia
gene replacement therapy
gene editing therapy
rna therapy
Online Access:https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.03.015

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https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.03.015

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