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Keywords = alpha-1-antitrypsin deficit

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29 pages, 2523 KB  
Review
Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?
by Lucía Bañuls, Daniel Pellicer, Silvia Castillo, María Mercedes Navarro-García, María Magallón, Cruz González and Francisco Dasí
J. Clin. Med. 2020, 9(8), 2577; https://doi.org/10.3390/jcm9082577 - 8 Aug 2020
Cited by 27 | Viewed by 8024
Abstract
Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and currently without curative treatment. After five decades of progress, many different vectors and gene editing tools for genetic engineering are now available. However, we are still a long way [...] Read more.
Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and currently without curative treatment. After five decades of progress, many different vectors and gene editing tools for genetic engineering are now available. However, we are still a long way from achieving a safe and efficient approach to gene therapy application in clinical practice. Here, we review three of the most common rare respiratory conditions—cystic fibrosis (CF), alpha-1 antitrypsin deficiency (AATD), and primary ciliary dyskinesia (PCD)—alongside attempts to develop genetic treatment for these diseases. Since the 1990s, gene augmentation therapy has been applied in multiple clinical trials targeting CF and AATD, especially using adeno-associated viral vectors, resulting in a good safety profile but with low efficacy in protein expression. Other strategies, such as non-viral vectors and more recently gene editing tools, have also been used to address these diseases in pre-clinical studies. The first gene therapy approach in PCD was in 2009 when a lentiviral transduction was performed to restore gene expression in vitro; since then, transcription activator-like effector nucleases (TALEN) technology has also been applied in primary cell culture. Gene therapy is an encouraging alternative treatment for these respiratory diseases; however, more research is needed to ensure treatment safety and efficacy. Full article
(This article belongs to the Special Issue Rare Respiratory Diseases: A Personal and a Public Health Problem)
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