Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer
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, an...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2019-02-01
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| Series: | Journal of Cardiovascular Development and Disease |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2308-3425/6/1/8 |
| _version_ | 1829519647470256128 |
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| author | Michael G. Katz Anthony S. Fargnoli Sarah M. Gubara Kenneth Fish Thomas Weber Charles R. Bridges Roger J. Hajjar Kiyotake Ishikawa |
| author_facet | Michael G. Katz Anthony S. Fargnoli Sarah M. Gubara Kenneth Fish Thomas Weber Charles R. Bridges Roger J. Hajjar Kiyotake Ishikawa |
| author_sort | Michael G. Katz |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-16T14:42:54Z |
| format | Article |
| id | doaj.art-eba4073123d04bceba63aab9a23938bf |
| institution | Directory Open Access Journal |
| issn | 2308-3425 |
| language | English |
| last_indexed | 2024-12-16T14:42:54Z |
| publishDate | 2019-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Cardiovascular Development and Disease |
| spelling | doaj.art-eba4073123d04bceba63aab9a23938bf2022-12-21T22:27:51ZengMDPI AGJournal of Cardiovascular Development and Disease2308-34252019-02-0161810.3390/jcdd6010008jcdd6010008Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene TransferMichael G. Katz0Anthony S. Fargnoli1Sarah M. Gubara2Kenneth Fish3Thomas Weber4Charles R. Bridges5Roger J. Hajjar6Kiyotake Ishikawa7Cardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USACardiovascular Research Center, Department of Cardiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USAAdvances 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.https://www.mdpi.com/2308-3425/6/1/8gene therapylung cellular structurebiological barriersviral vectorsendotracheal route of deliveryhereditarymutation |
| spellingShingle | Michael G. Katz Anthony S. Fargnoli Sarah M. Gubara Kenneth Fish Thomas Weber Charles R. Bridges Roger J. Hajjar Kiyotake Ishikawa Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer Journal of Cardiovascular Development and Disease gene therapy lung cellular structure biological barriers viral vectors endotracheal route of delivery hereditary mutation |
| title | Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer |
| title_full | Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer |
| title_fullStr | Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer |
| title_full_unstemmed | Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer |
| title_short | Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer |
| title_sort | targeted gene delivery through the respiratory system rationale for intratracheal gene transfer |
| topic | gene therapy lung cellular structure biological barriers viral vectors endotracheal route of delivery hereditary mutation |
| url | https://www.mdpi.com/2308-3425/6/1/8 |
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