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Article

Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia.

Human gene therapy - 1 Dec 2025

Huo Chunxiao, Luo Ting, Wu Lei, Yang Feng, Xu Zhangqi, Tao Xiaofen, Xia Junhua, Zhou Tianhua, Jiang Yuan, Xie Shanshan

Abstract excerpt

Primary ciliary dyskinesia (PCD) is a genetic disorder characterized by defective ciliary motility, leading to recurrent respiratory infections and chronic airway damage. Gene therapy holds promise for treating PCD, but its effectiveness in patient-derived models remains uncertain. This study aimed to evaluate the therapeutic potential of lentiviral gene delivery in restoring ciliary function in patient-derived...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 40947900
DOI: 10.1177/10430342251378128

Genes

DNAAF1
DNAAF3
DNAAF6

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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