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Gene therapy for type 1 diabetes

Is it ready for the clinic?

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Abstract

This review, in addition to updating the growing list of type 1 diabetes-relevant gene therapies, offers an outline of short-term objectives that can readily be met to move, at least, adenoviral and adeno-associated viral-based protocols into the clinic, first as a means of facilitating islet allografts as well as platforms with which to introduce immunoregulatory transgenes. A wide array of genes have been tested to restore insulin production, to drive the differentiation of insulin-producing progenitors, and to confer immunosuppression in an antigen-and tissue-restricted manner.

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Author notes

  1. Nick Giannoukakis PhD

    Present address: Department of Pathology, University of Pittsburgh School of Medicine, Diabetes Institute, Rangos Research Center, 3460 Fifth Avenue, 15213, Pittsburgh, PA

Authors and Affiliations

  1. Diabetes Institute, Children's Hospital of Pittsburgh, Pittsburgh, PA, USA

    Antonella D'Anneo, Pleunie Rood, Rita Bottino, A. N. Balamurugan, Jing He & Nick Giannoukakis PhD

  2. Department of Pediatrics, Division of Immunogenetics, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

    Antonella D'Anneo, Pleunie Rood, Rita Bottino, A. N. Balamurugan & Jing He

  3. Department of Biochemical Sciences, University of Palermo, Palermo, Italy

    Antonella D'Anneo

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  1. Antonella D'Anneo
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  2. Pleunie Rood
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D'Anneo, A., Rood, P., Bottino, R. et al. Gene therapy for type 1 diabetes. Immunol Res 36, 83–89 (2006). https://doi.org/10.1385/IR:36:1:83

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