Cell and gene therapy for kidney disease

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).

Article  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

Wheeler, D. C. & Steiger, J. Evolution and etiology of cardiovascular diseases in renal transplant recipients. Transplantation 70, Ss41–Ss45 (2000).

CAS  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

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).

Article  Google Scholar 

Jayasinghe, K. et al. Clinical impact of genomic testing in patients with suspected monogenic kidney disease. Genet. Med. 23, 183–191 (2021).

Article  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

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).

Article  CAS  Google Scholar 

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).

Article  PubMed  Google Scholar 

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).

Article  PubMed  Google Scholar 

Tögel, F. E. & Westenfelder, C. Mesenchymal stem cells: a new therapeutic tool for AKI. Nat. Rev. Nephrol. 6, 179–183 (2010).

Article  PubMed 

留言 (0)

沒有登入
gif