title: Gene based therapies for kidney regeneration.
abstract: In this review we provide an overview of the expanding molecular toolbox that is available for gene based therapies and how these therapies can be used for a large variety of kidney diseases. Gene based therapies range from restoring gene function in genetic kidney diseases to steering complex molecular pathways in chronic kidney disorders, and can provide a treatment or cure for diseases that otherwise may not be targeted. This approach involves the delivery of recombinant DNA sequences harboring therapeutic genes to improve cell function and thereby promote kidney regeneration. Depending on the therapy, the recombinant DNA will express a gene that directly plays a role in the function of the cell (gene addition), that regulates the expression of an endogenous gene (gene regulation), or that even changes the DNA sequence of endogenous genes (gene editing). Some interventions involve permanent changes in the genome whereas others are only temporary and leave no trace. Efficient and safe delivery are important steps for all gene based therapies and also depend on the mode of action of the therapeutic gene. Here we provide examples on how the different methods can be used to treat various diseases, which technologies are now emerging (such as gene repair through CRISPR/Cas9) and what the opportunities, perspectives, potential and the limitations of these therapies are for the treatment of kidney diseases.
authors.affiliation: Div Pharmacology, Department of Pharmaceutical Sciences, Utrecht Institute for Pharmaceutical Sciences, University of Utrecht, The Netherlands. Electronic address: m.j.janssen1@uu.nl.
authors.forename: Manoe J
authors.initials: MJ
authors.lastname: Janssen
authors.affiliation: Department of Development and Regeneration, Organ System Cluster, Group of Biomedical Sciences, KU Leuven, Belgium.
authors.forename: Fanny O
authors.initials: FO
authors.lastname: Arcolino
authors.affiliation: Div Pharmacology, Department of Pharmaceutical Sciences, Utrecht Institute for Pharmaceutical Sciences, University of Utrecht, The Netherlands.
authors.forename: Perry
authors.initials: P
authors.lastname: Schoor
authors.affiliation: Department of Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences, University of Utrecht, The Netherlands.
authors.forename: Robbert Jan
authors.initials: RJ
authors.lastname: Kok
authors.affiliation: Department of Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences, University of Utrecht, The Netherlands.
authors.forename: Enrico
authors.initials: E
authors.lastname: Mastrobattista
mesh_terms: D000818:Animals; D004337:Drug Carriers / Q000737:chemistry; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D007668:Kidney / Q000502:physiology*; D012038:Regeneration / Q000235:genetics*
chemical_list: D004337:Drug Carriers
keywords: CRISPR/Cas9; Gene therapy; Kidney disease; Viral and non-viral vectors