Evans, M. et al. A narrative review of chronic kidney disease in clinical practice: current challenges and future perspectives. Adv. Ther. 39, 33–43 (2022).
Hill, N. R. et al. Global prevalence of chronic kidney disease–a systematic review and meta-analysis. PLoS ONE 11, e0158765 (2016).
Article PubMed PubMed Central Google Scholar
Levey, A. S. & Coresh, J. Chronic kidney disease. Lancet 379, 165–180 (2012).
United States Renal Data System. USRDS Annual Data Report: Epidemiology of Kidney Disease in the United States (National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, 2021).
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M. & Abedi, M. R. Gene therapy clinical trials worldwide to 2017: an update. J. Gene Med. 20, e3015 (2018).
Wheeler, D. C. & Steiger, J. Evolution and etiology of cardiovascular diseases in renal transplant recipients. Transplantation 70, Ss41–Ss45 (2000).
Hildebrandt, F. Genetic kidney diseases. Lancet 375, 1287–1295 (2010).
Article CAS PubMed PubMed Central Google Scholar
Schrezenmeier, E. et al. The underestimated burden of monogenic kidney disease in adults waitlisted for kidney transplantation. Genet. Med. 23, 1219–1224 (2021).
Article CAS PubMed PubMed Central Google Scholar
Imai, E., Takabatake, Y., Mizui, M. & Isaka, Y. Gene therapy in renal diseases. Kidney Int. 65, 1551–1555 (2004).
Article CAS PubMed Google Scholar
Armstrong, M. E. & Thomas, C. P. Diagnosis of monogenic chronic kidney diseases. Curr. Opin. Nephrol. Hypertens. 28, 183–194 (2019).
Connaughton, D. M. et al. Monogenic causes of chronic kidney disease in adults. Kidney Int. 95, 914–928 (2019).
Article PubMed PubMed Central Google Scholar
KDIGO Conference Participants. Genetics in chronic kidney disease: conclusions from a kidney disease: improving global outcomes (KDIGO) controversies conference. Kidney Int. 101, 1126–1141 (2022).
Jayasinghe, K. et al. Clinical impact of genomic testing in patients with suspected monogenic kidney disease. Genet. Med. 23, 183–191 (2021).
Groopman, E. E. et al. Diagnostic utility of exome sequencing for kidney disease. New Engl. J. Med. 380, 142–151 (2019).
Article CAS PubMed Google Scholar
Moore, D. F., Ries, M., Forget, E. L. & Schiffmann, R. Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry–Anderson disease. Pharmacoeconomics 25, 201–208 (2007).
Oder, D., Nordbeck, P. & Wanner, C. Long term treatment with enzyme replacement therapy in patients with fabry disease. Nephron 134, 30–36 (2016).
Article CAS PubMed Google Scholar
Rohrbach, M. & Clarke, J. T. Treatment of lysosomal storage disorders: progress with enzyme replacement therapy. Drugs 67, 2697–2716 (2007).
Article CAS PubMed Google Scholar
Grange, C. & Bussolati, B. Extracellular vesicles in kidney disease. Nat. Rev. Nephrol. 18, 499–513 (2022).
Article PubMed PubMed Central Google Scholar
Biancone, L. & Camussi, G. Potential use of stem or progenitor cells for kidney regeneration. Nat. Rev. Nephrol. 10, 67–68 (2014).
Article CAS PubMed Google Scholar
Bussolati, B. & Camussi, G. Therapeutic use of human renal progenitor cells for kidney regeneration. Nat. Rev. Nephrol. 11, 695–706 (2015).
Article CAS PubMed Google Scholar
Couzin, J. & Kaiser, J. Gene therapy. As Gelsinger case ends, gene therapy suffers another blow. Science 307, 1028 (2005).
Article CAS PubMed Google Scholar
Brunetti-Pierri, N. & Ng, P. Helper-dependent adenoviral vectors for liver-directed gene therapy. Hum. Mol. Genet. 20, R7–R13 (2011).
Article CAS PubMed PubMed Central Google Scholar
Piccolo, P. & Brunetti-Pierri, N. Challenges and prospects for helper-dependent adenoviral vector-mediated gene therapy. Biomedicines 2, 132–148 (2014).
Article PubMed PubMed Central Google Scholar
Atchison, R. W., Casto, B. C. & Hammon, W. M. Adenovirus-associated defective virus particles. Science 149, 754–756 (1965).
Article CAS PubMed Google Scholar
Blaese, R. M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270, 475–480 (1995).
Article CAS PubMed Google Scholar
Hacein-Bey-Abina, S. et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New Engl. J. Med. 346, 1185–1193 (2002).
Article CAS PubMed Google Scholar
Hacein-Bey-Abina, S. et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. New Engl. J. Med. 348, 255–256 (2003).
Hacein-Bey-Abina, S. et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415–419 (2003).
Article CAS PubMed Google Scholar
Philippidis, A. Brother of cure rare disease CEO dies in trial of Duchenne muscular dystrophy therapy. Hum. Gene Ther. 33, 1224–1227 (2022).
Article CAS PubMed Google Scholar
Barrett, D. et al. Gene, Cell, and RNA Therapy Landscape: Q3 2022 Quarterly Data Report, https://asgct.org/global/documents/asgct-citeline-q3-2022-report.aspx (2022).
Moran, N. First gene therapy approved. Nat. Biotechnol. 30, 1153 (2012).
Braendstrup, P., Levine, B. L. & Ruella, M. The long road to the first FDA-approved gene therapy: chimeric antigen receptor T cells targeting CD19. Cytotherapy 22, 57–69 (2020).
Article CAS PubMed PubMed Central Google Scholar
Bishop, D. C. et al. Development of CAR T-cell lymphoma in two of ten patients effectively treated with piggyBac modified CD19 CAR T-cells. Blood 138, 1504–1509 (2021).
Article CAS PubMed Google Scholar
Micklethwaite, K. P. et al. Investigation of product derived lymphoma following infusion of piggyBac modified CD19 chimeric antigen receptor T-cells. Blood 138, 1391–1405 (2021).
Article CAS PubMed PubMed Central Google Scholar
Saleem, M. A. Molecular stratification of idiopathic nephrotic syndrome. Nat. Rev. Nephrol. 15, 750–765 (2019).
Juhila, J. et al. Inducible nephrin transgene expression in podocytes rescues nephrin-deficient mice from perinatal death. Am. J. Pathol. 176, 51–63 (2010).
Article CAS PubMed PubMed Central Google Scholar
Naylor, R. W., Morais, M. & Lennon, R. Complexities of the glomerular basement membrane. Nat. Rev. Nephrol. 17, 112–127 (2021).
Article CAS PubMed Google Scholar
Lin, X., Suh, J. H., Go, G. & Miner, J. H. Feasibility of repairing glomerular basement membrane defects in Alport syndrome. J. Am. Soc. Nephrol. 25, 687–692 (2014).
Article CAS PubMed Google Scholar
Dong, K. et al. Renal plasticity revealed through reversal of polycystic kidney disease in mice. Nat. Genet. 53, 1649–1663 (2021).
Article CAS PubMed PubMed Central Google Scholar
Chebib, F. T. & Torres, V. E. Autosomal dominant polycystic kidney disease: core curriculum 2016. Am. J. Kidney Dis. 67, 792–810 (2016).
Tögel, F. E. & Westenfelder, C. Mesenchymal stem cells: a new therapeutic tool for AKI. Nat. Rev. Nephrol. 6, 179–183 (2010).
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