File:40265 2022 1735 Fig3 HTML.webp
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40265_2022_1735_Fig3_HTML.webp (685 × 468 pixels, file size: 53 KB, MIME type: image/webp)
Summary
Description |
English: Strategies for molecular therapy in primary hyperoxaluria (updated from Martin-Higueras et al. [7]): gene therapy with single-stranded adeno-associated virus (ssAAV) carrying one copy of AGXT cDNA (here also applicable SV40 as a vehicle); cell therapy by hepatocyte transplantation, including the potential autologous transplantation of human induced pluripotent stem cell-derived hepatocytes; proteostasis regulation therapy targeting molecular chaperones (Hsp60 and Hsp90) such as dequalinium chloride, monesin, and emetine, or directly stabilizing the (AGT) enzyme with the cofactor pyridoxine (B6); enzyme replacement therapy (ERT) by delivery of polymer-conjugated AGT proteins into the peroxisomal compartment; and substrate reduction therapy (SRT) through inhibition of glycolate oxidase (GO) in the peroxisome and/or lactate dehydrogenase A (LDHA) in the cytosol either by RNA interference or by small molecules, or by editing the corresponding gene. AGT alanine:glyoxylate aminotransferase, responsible for PH1, AGT-Mi AGT in the minor haplotype, DAO D-amino acid oxidase, GRHPR glyoxylate reductase/hydroxypyruvate reductase, enzyme deficient in PH2, HOGA1 4-hydroxy-2-oxoglutarate aldolase 1, involved in PH3, LDH L-lactate dehydrogenase |
Date | |
Source | https://link.springer.com/article/10.1007/s40265-022-01735-x |
Author | Bernd Hoppe & Cristina Martin-Higueras |
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English: This file is licensed CC BY-NC 4.0
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