Structural insights of Adeno-Associated virus 5. A gene therapy Vector for Cystic Fibrosis

NIAID Data Ecosystem2026-05-01 收录

病毒载体结构解析

囊性纤维化基因治疗

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Structural insights of Adeno-Associated virus 5. A gene therapy Vector for Cystic Fibrosis

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2023-09-06

Functional Correction of CFTR Mutations in Human Airway Epithelial Cells using Adenine Base Editors

囊性纤维化基因治疗

碱基编辑

We investigated the use of adenine base editor (ABE) ribonucleoproteins (RNPs) that convert A-T to G-C base pairs as a therapeutic strategy for three cystic fibrosis disease causing mutations. Using A

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Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing.. Homo sapiens

CFTR剪接突变修复

囊性纤维化基因治疗

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene. The 3272-26A>G and 3849+10kbC>T CFTR mutations alter the correct splicing of the CFTR gene, generating new

NIAID Data Ecosystem40

Bronchoalveolar Fluid Is Not a Major Hindrance to Virus-Mediated Gene Therapy in Cystic Fibrosis

囊性纤维化基因治疗

病毒载体递送优化

Successfully targeting the airway epithelium is essential for gene therapy of some pulmonary diseases. However, the airway epithelium is resistant to virus-mediated gene transfer with commonly used ve

PubMed Central10

Inflammatory program of CF-TG cells transfected with cftr gene.

囊性纤维化基因治疗

炎症基因表达

The expression levels of seven commonly regulated genes were determined by qRT-PCR. The results are expressed as the fold changes between CF-TG cells and wt TG cells, and between CF-TG cells transfect

Figshare2015-12-02 更新40

MOESM11 of Gene therapy-emulating small molecule treatments in cystic fibrosis airway epithelial cells and patients

囊性纤维化基因治疗

囊性纤维化药物转录组学

Additional file 11. Genes downregulated by digitoxin treatment and gene therapy against IB3-1 controls annotated to inflammatory ontologies, compared in parallel to effects of VX-661, VX-770 and VX-80

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Structural insights of Adeno-Associated virus 5. A gene therapy Vector for Cystic Fibrosis

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