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Improving Molecular Therapy in the Kidney

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Abstract

Mutations in approximately 80 genes have been implicated as the cause of various genetic kidney diseases. However, gene delivery to kidney cells from the blood is inefficient because of the natural filtering functions of the glomerulus, and research into and development of gene therapy directed toward kidney disease has lagged behind as compared with hepatic, neuromuscular, and ocular gene therapy. This lack of progress is in spite of numerous genetic mouse models of human disease available to the research community and many vectors in existence that can theoretically deliver genes to kidney cells with high efficiency. In the past decade, several groups have begun to develop novel injection techniques in mice, such as retrograde ureter, renal vein, and direct subcapsular injections to help resolve the issue of gene delivery to the kidney through the blood. In addition, the ability to retarget vectors specifically toward kidney cells has been underutilized but shows promise. This review discusses how recent advances in gene delivery to the kidney and the field of gene therapy can leverage the wealth of knowledge of kidney genetics to work toward developing gene therapy products for patients with kidney disease.

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Authors and Affiliations

  1. Virology and Gene Therapy Graduate Program, Mayo Clinic, Rochester, MN, USA

    Jeffrey D. Rubin

  2. Division of Infectious Diseases, Department of Internal Medicine, Mayo Clinic, 200 First Street SW, Rochester, MN, USA

    Michael A. Barry

  3. Department of Immunology, Mayo Clinic, Rochester, MN, USA

    Michael A. Barry

  4. Department of Molecular Medicine, Mayo Clinic, Rochester, MN, USA

    Michael A. Barry

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  1. Jeffrey D. Rubin
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Correspondence to Michael A. Barry.

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This work was supported by the Department of Molecular Medicine at Mayo Clinic (J.D.R.), National Institute of Diabetes Digestive and Kidney Diseases Grant number 1F31DK123858–01 and P30-DK090728 (J.D.R.), and the Department of Laboratory Medicine and Pathology at Mayo Clinic (M.A.B.).

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Jeffrey D. Rubin and Michael A. Barry have no conflicts of interest that are directly relevant to the content of this article.

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Rubin, J.D., Barry, M.A. Improving Molecular Therapy in the Kidney. Mol Diagn Ther 24, 375–396 (2020). https://doi.org/10.1007/s40291-020-00467-6

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