Assessing the potential of <i>DZIP1L</i> gene in autosomal recessive polycystic kidney disease gene therapy

Fahreddin Palaz

doi:10.1002/pdi3.44

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Pediatric Discovery ›› 2024, Vol. 2 ›› Issue (1) : e44. DOI: 10.1002/pdi3.44

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Assessing the potential of DZIP1L gene in autosomal recessive polycystic kidney disease gene therapy

Fahreddin Palaz¹^,²

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Keywords

AAV / ARPKD / DZIP1L / gene therapy / PKHD1

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Fahreddin Palaz. Assessing the potential of DZIP1L gene in autosomal recessive polycystic kidney disease gene therapy. Pediatric Discovery, 2024, 2(1): e44 https://doi.org/10.1002/pdi3.44

References

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[1]	Lu Y, Song Y, Sun S, Zhang L, Chen Y. Gene therapy for pediatric genetic kidney diseases. Pediatr Discov. 2023;1(1):e16.

[2]	Lu H, Galeano MCR, Ott E, et al. Mutations in DZIP1L, which encodes a ciliary-transition-zone protein, cause autosomal recessive polycystic kidney disease. Nat Genet. 2017;49(7):1025-1034.

[3]	Bergmann C, Guay-Woodford LM, Harris PC, Horie S, Peters DJM, Torres VE. Polycystic kidney disease. Nat Rev Dis Prim. 2018;4(1):50.

[4]	Hertz JM, Svenningsen P, Dimke H, et al. Detection of DZIP1L mutations by whole-exome sequencing in consanguineous families with polycystic kidney disease. Pediatr Nephrol. 2022;37(11):2657-2665.

[5]	Cordido A, Vizoso-Gonzalez M, Garcia-Gonzalez MA. Molecular pathophysiology of autosomal recessive polycystic kidney disease. Int J Mol Sci. 2021;22(12):6523.

[6]	Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18(5):358-378.

[7]	Rubin JD, Barry MA. Improving molecular therapy in the kidney. Mol Diagn Ther. 2020;24(4):375-396.

[8]	WareJoncas Z, Campbell JM, Martínez-Gálvez G, et al. Precision gene editing technology and applications in nephrology. Nat Rev Nephrol. 2018;14(11):663-677.

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