Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease.

Human Gene Therapy
Yinghua TangJ F Engelhardt

Abstract

After more than two decades since clinical trials tested the first use of recombinant adeno-associated virus (rAAV) to treat cystic fibrosis (CF) lung disease, gene therapy for this disorder has undergone a tremendous resurgence. Fueling this enthusiasm has been an enhanced understanding of rAAV transduction biology and cellular processes that limit transduction of airway epithelia, the development of new rAAV serotypes and other vector systems with high-level tropism for airway epithelial cells, an improved understanding of CF lung pathogenesis and the cellular targets for gene therapy, and the development of new animal models that reproduce the human CF disease phenotype. These advances have created a preclinical path for both assessing the efficacy of gene therapies in the CF lung and interrogating the target cell types in the lung required for complementation of the CF disease state. Lessons learned from early gene therapy attempts with rAAV in the CF lung have guided thinking for the testing of next-generation vector systems. Although unknown questions still remain regarding the cellular targets in the lung that are required or sufficient to complement CF lung disease, the field is now well positioned to tackle these chall...Continue Reading

References

Jan 1, 1980·Cell and Tissue Research·R J PackJ G Widdicombe
Feb 1, 1994·The Journal of Clinical Investigation·J F EngelhardtJ M Wilson
Apr 1, 1994·American Journal of Respiratory Cell and Molecular Biology·E F TizzanoM Buchwald
Aug 11, 1995·Science·M J StuttsR C Boucher
Jun 1, 1994·American Journal of Respiratory Cell and Molecular Biology·R R MercerJ D Crapo
Aug 26, 1998·Proceedings of the National Academy of Sciences of the United States of America·L ZhangJ Y Dong
Oct 22, 1998·European Journal of Human Genetics : EJHG·Q Jiang, J F Engelhardt
Jan 29, 1999·Physiological Reviews·B R Grubb, R C Boucher
Jun 7, 2000·The Journal of Clinical Investigation·D DuanJ F Engelhardt
Dec 16, 2000·Journal of Anatomy·H K ChoiJ H Widdicombe
Feb 13, 2001·American Journal of Respiratory Cell and Molecular Biology·X WangJ F Engelhardt
Mar 29, 2001·Journal of Anatomy·J H WiddicombeS J Bastacky
Jan 30, 2002·The Journal of General Physiology·X FangM A Matthay
Feb 21, 2002·Proceedings of the National Academy of Sciences of the United States of America·Lynda S OstedgaardMichael J Welsh
Dec 12, 2003·Proceedings of the National Academy of Sciences of the United States of America·Raymond D CoakleyRichard C Boucher
Nov 27, 2004·Current Topics in Developmental Biology·Xiaoming LiuJohn F Engelhardt
Nov 27, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Liang N ZhangJohn F Engelhardt
Feb 11, 2005·Proceedings of the National Academy of Sciences of the United States of America·Lynda S OstedgaardMichael J Welsh
Feb 18, 2005·Molecular Biology of the Cell·Silvia M KredaRichard C Boucher
Mar 5, 2005·American Journal of Respiratory Cell and Molecular Biology·Rana DajaniJohn F Engelhardt
Apr 15, 2005·Gene Therapy·W DingJ F Engelhardt
Aug 23, 2005·Proceedings of the American Thoracic Society·Jeffrey J Wine, Nam Soo Joo
Oct 14, 2005·Proceedings of the American Thoracic Society·Michael A MatthayXiaohui Fang
Jan 24, 2006·Nature Biotechnology·Narendra MaheshriDavid V Schaffer
Jan 31, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Wei DingJohn F Engelhardt

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Citations

Jul 29, 2020·International Journal of Molecular Sciences·Pascal DuchesneauGolnaz Karoubi
Jan 21, 2021·Molecular Therapy : the Journal of the American Society of Gene Therapy·Soon H Choi, John F Engelhardt
Nov 20, 2020·Molecular Therapy. Methods & Clinical Development·Yinghua TangJohn F Engelhardt

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Methods Mentioned

BETA
scRNAseq

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