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Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer

Cardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
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J. Cardiovasc. Dev. Dis. 2019, 6(1), 8; https://doi.org/10.3390/jcdd6010008
Received: 19 December 2018 / Revised: 11 February 2019 / Accepted: 13 February 2019 / Published: 15 February 2019
(This article belongs to the Special Issue Cardiaovascular Gene Therapy)
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Abstract

Advances in DNA- and RNA-based technologies have made gene therapy suitable for many lung diseases, especially those that are hereditary. The main objective of gene therapy is to deliver an adequate amount of gene construct to the intended target cell, achieve stable transduction in target cells, and to produce a clinically therapeutic effect. This review focuses on the cellular organization in the normal lung and how gene therapy targets the specific cell types that are affected by pulmonary disorders caused by genetic mutations. Furthermore, it examines the pulmonary barriers that can compromise the absorption and transduction of viral vectors and genetic agents by the lung. Finally, it discusses the advantages and limitations of direct intra-tracheal gene delivery with different viral vectors in small and large animal models and in clinical trials. View Full-Text
Keywords: gene therapy; lung cellular structure; biological barriers; viral vectors; endotracheal route of delivery; hereditary; mutation gene therapy; lung cellular structure; biological barriers; viral vectors; endotracheal route of delivery; hereditary; mutation
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Katz, M.G.; Fargnoli, A.S.; Gubara, S.M.; Fish, K.; Weber, T.; Bridges, C.R.; Hajjar, R.J.; Ishikawa, K. Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer. J. Cardiovasc. Dev. Dis. 2019, 6, 8.

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