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Do we need marker gene studies in humans to improve clinical AAV gene therapy?

Gene Therapy volume 24, pages 72–73 (2017)Cite this article

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In April 1990, the Chicago Tribune published a front page article entitled ‘Gene therapy poised to reinvent medicine’. More than a quarter of a century later, even the most bullish scientists working in the field of gene therapy would probably acknowledge that ‘the reinvention of medicine’ by gene therapy has yet to be realized. Nonetheless, I think it is fair to say that we are at the cusp of using somatic gene transfer to treat, if not cure, numerous genetic and even acquired diseases. At the same time, it would be foolish to deny that formidable obstacles remain to realize fully the great potential that gene therapy provides to revolutionize the treatment of monogeneic Mendelian diseases and to use therapeutic gene transfer as an important treatment modality for complex genetic disorders and acquired diseases.

From its inception, the most daunting roadblock to successful therapeutic gene transfer has been the efficient and selective delivery of the therapeutic gene to the desired target organ(s). As Inder Verma from the Salk Institute put it: ‘There are only three problems in gene therapy, delivery, delivery and delivery’.1

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Acknowledgements

I would like to thank Els Henckaerts, Michael Linden, Fadi Akar and Roger Hajjar for stimulating discussions. This work is supported by R01 HL131404 and a Trans-Atlantic Network of Excellence grant (14CVD03) from the Leducq Foundation.

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  1. Department of Medicine, Division of Cardiology, Cardiovascular Research Center and Graduate School of Biomedical Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    T Weber

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Correspondence to T Weber.

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Weber, T. Do we need marker gene studies in humans to improve clinical AAV gene therapy?. Gene Ther 24, 72–73 (2017). https://doi.org/10.1038/gt.2016.84

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