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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Autors principals:	LU Wanqing, LI Yixuan, HE Miao, TIAN Xinlun, LIU Yaping
Format:	Artigo
Idioma:	Chinês
Publicat:	Editorial Office of Journal of Rare Diseases 2025-07-01
Col·lecció:	罕见病研究
Matèries:	primary ciliary dyskinesia gene replacement therapy gene editing therapy rna therapy
Accés en línia:	https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.03.015
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