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Review

Adeno-Associated Virus (AAV) gene therapy for cystic fibrosis: current barriers and recent developments

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Pages 1265-1273 | Received 28 Feb 2017, Accepted 23 Jun 2017, Published online: 06 Jul 2017

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John W. Hanrahan, Yukiko Sato, Graeme W. Carlile, Gregor Jansen, Jason C. Young & David Y. Thomas. (2019) Cystic Fibrosis: Proteostatic correctors of CFTR trafficking and alternative therapeutic targets.. Expert Opinion on Therapeutic Targets 23:8, pages 711-724.
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ANSELM J. Gadenstaetter, LUKAS SCHMUTZLER, DIRK GRIMM & LUKAS D. LANDEGGER. (2022) Intranasal application of adeno-associated viruses: a systematic review. Translational Research 248, pages 87-110.
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Uday K Baliga & David A Dean. (2020) Pulmonary gene delivery—Realities and possibilities. Experimental Biology and Medicine 246:3, pages 260-274.
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Yanerys Colon-Cortes, Mutasim Abu Hasan & George Aslanidi. (2020) Intra-tracheal delivery of AAV6 vectors results in sustained transduction in murine lungs without genomic integration. Gene 763, pages 100037.
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Zhichang Peter Zhou, Liang Leo Yang, Huibi Cao, Ziyan Rachel Chen, Yiqian Zhang, Xiao-Yan WenJim Hu. (2019) In Vitro Validation of a CRISPR-Mediated CFTR Correction Strategy for Preclinical Translation in Pigs . Human Gene Therapy 30:9, pages 1101-1116.
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Ren-Yuan Bai, Dominic Esposito, Ada J. Tam, Frank McCormick, Gregory J. Riggins, D. Wade Clapp & Verena Staedtke. (2019) Feasibility of using NF1-GRD and AAV for gene replacement therapy in NF1-associated tumors. Gene Therapy 26:6, pages 277-286.
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Bradley A. Hamilton, Xiaopeng Li, Alejandro A. Pezzulo, Mahmoud H. Abou Alaiwa & Joseph Zabner. (2019) Polarized AAVR expression determines infectivity by AAV gene therapy vectors. Gene Therapy 26:6, pages 240-249.
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Antonio Alberto Rodríguez Sousa, Jesús M. Barandica Fernández, Jonathan Miller & Matías Mir-Montejano. (2018) Physiological study of pulmonary involvement in adults with cystic fibrosis through simulated modeling of different clinical scenarios. Medical & Biological Engineering & Computing 57:2, pages 413-425.
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Martin Donnelley & David W. Parsons. (2018) Gene Therapy for Cystic Fibrosis Lung Disease: Overcoming the Barriers to Translation to the Clinic. Frontiers in Pharmacology 9.
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Miquéias Lopes-Pacheco, Jamil Z. Kitoko, Marcelo M. Morales, Hilda Petrs-Silva & Patricia R.M. Rocco. (2018) Self-complementary and tyrosine-mutant rAAV vectors enhance transduction in cystic fibrosis bronchial epithelial cells. Experimental Cell Research 372:2, pages 99-107.
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Ema Robinson, Kelvin D. MacDonald, Kai Slaughter, Madison McKinney, Siddharth Patel, Conroy Sun & Gaurav Sahay. (2018) Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis. Molecular Therapy 26:8, pages 2034-2046.
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Massimo Fioranelli & Alireza Sepehri. (2018) A mathematical model for the virus medical imaging technique. International Journal of Geometric Methods in Modern Physics 15:07, pages 1850121.
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Marne C. Hagemeijer, Daniel J. Siegwart, Lisa J. Strug, Liudmila Cebotaru, Michael J. Torres, Aderonke Sofoluwe & Jeffrey M. Beekman. (2018) Translational research to enable personalized treatment of cystic fibrosis. Journal of Cystic Fibrosis 17:2, pages S46-S51.
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William B. GugginoJanet BensonJeanClare SeagraveZiying YanJohn EngelhardtGuangping GaoThomas J. ConlonLiudmila Cebotaru. (2017) A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System. Human Gene Therapy Clinical Development 28:3, pages 145-156.
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