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March 28, 2024Molecular Therapy — Methods & Clinical DevelopmentOpen Access

Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs

Yinghua Tang Mehrnoosh Ebadi Junying Lei Zehua Feng Shahab Fakhari Peipei Wu Mark Bodman‐Smith Maria P. Limberis Roland Kolbeck Katherine J. D. A. Excoffon Ziying Yan John F. Engelhardt

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Abstract

The dosing interval for effective recombinant adeno-associated virus (rAAV)-mediated gene therapy of cystic fibrosis lung disease remains unknown. Here, we assessed the durability of rAAV2.5T-fCFTRΔR-mediated transgene expression and neutralizing antibody (NAb) responses in lungs of adult wild-type ferrets. Within the first 3 months following rAAV2.5T-fCFTRΔR delivery to the lung,

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Cite This Study

Tang et al. (2024) studied this question.

synapsesocial.com/papers/68e71edfb6db643587698a4b https://doi.org/10.1016/j.omtm.2024.101244

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Durational Study of Persistent Transduction of Rhesus Macaque Lung and Other Organs Following Single Dosing with AAV1-CFTROriginal Research · 2026
2Multilineage transduction of resident lung cells in vivo by AAV2/8 for α1-antitrypsin gene therapypreclinical · 2016 · 14 citations
3Repeat Transduction in the Mouse Lung by Using Adeno-Associated Virus Vectors with Different SerotypesOriginal Research · 2000 · 258 citations
4Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction propertiespreclinical · 2023 · 12 citations
5Inhalation of SP-101 followed by inhaled doxorubicin results in robust and durable hCFTRΔR transgene expression in the airways of wild-type and cystic fibrosis ferrets2024 · 13 citations

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