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Treating genes and patients

Gene Therapy volume 27, pages 109–110 (2020)Cite this article

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The Original Article was published on 12 July 2019

Our understanding of the clinical impact of gene mutation is growing rapidly and has opened a range of possible avenues for therapy of genetic disease. An example in point is the most common, life-limiting mutation in Caucasian populations, being the cystic fibrosis (CF) gene [1]. In CF, there is now a stratification of specific mutations into classes that reflect the main intracellular events limiting gene-product function [2]. The growing list of CF transmembrane regulator protein (CFTR) gene mutations now re-defines the task of dealing with either (a) the gene itself (irrespective of mutation), or (b) classes of mutations with distinctly different characteristics and functional activity. In this edition of the journal, Schneider-Futschik examines the current status of gene therapy versus gene product modulation (Schneider-Futschik Ref here). Promising as they are, both approaches are treatments without offering a ‘cure’, in the way antibiotics help eliminate infection.

At present, there is no enduring cure for CF, until the question of germ-line correction has been addressed. Further, the inability to effectively treat early progenitors (except as happens in conditions such as ADA-SCID [3, 4]) means that repeated treatments of somatic cells must be maintained. Conceptually, healthy mesenchymal stem cells might be used to replace affected progenitors [5], resulting in an enduring response. Although some doubt exists over their homing capacity to reach and populate target organs [6], it is encouraging to be reminded of the natural experiment of lung transplantation, in which epithelial precursors migrate into the graft from the host and differentiate within weeks of the procedure [7].

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Authors and Affiliations

  1. Cystic Fibrosis Service, Alfred Health, Melbourne, Australia

    John Wilson

  2. Department of Medicine, Central Clinical School, Monash University, Melbourne, Australia

    John Wilson

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Correspondence to John Wilson.

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JW has received clinical trial support and consultancy funding from Vertex Pharmaceuticals Inc, as well as clinical trial support from Galapagos NV.

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Wilson, J. Treating genes and patients. Gene Ther 27, 109–110 (2020). https://doi.org/10.1038/s41434-019-0111-6

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