Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1

Kukreja, Anjli; Lasaro, Melissa A.; Cobaugh, Christian; Forbes, Chris D.; Tang, Jianping; Gao, Xiang; Martín-Higueras, Cristina; Pey, Ángel L.; Salido, Eduardo; Sobolov, Susan B.; Subramanian, Romesh R.

doi:10.1089/nat.2018.0740

Cited by 13 publications

(2 citation statements)

References 36 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Conjugating AGT with poly(ethylene glycol)-co-poly(L-glutamic acid) (PEG-PGA) block co-polymers to create PEG-PGA-AGT conjugates facilitates their smooth internalization and accurate subcellular localization for glyoxylate metabolism in PH1 cell models, while also addressing plasma stability and immunogenicity concerns [150]. AGT gene delivery employs recombinant adeno-associated virus (AAV) vectors with complementary DNA (cDNA) and lipid nanoparticles encapsulating mRNA, effectively reducing the UOx levels in AGTX gene knockout mouse models [151][152][153]. Indirect ERT primarily involves liver cell transplantation (LCT) with normal function, either autologous or allogeneic.…”

Section: Ertmentioning

confidence: 99%

Navigating the Evolving Landscape of Primary Hyperoxaluria: Traditional Management Defied by the Rise of Novel Molecular Drugs

Huang,

Zhu,

Zhou

et al. 2024

Biomolecules

View full text Add to dashboard Cite

Primary hyperoxalurias (PHs) are inherited metabolic disorders marked by enzymatic cascade disruption, leading to excessive oxalate production that is subsequently excreted in the urine. Calcium oxalate deposition in the renal tubules and interstitium triggers renal injury, precipitating systemic oxalate build-up and subsequent secondary organ impairment. Recent explorations of novel therapeutic strategies have challenged and necessitated the reassessment of established management frameworks. The execution of diverse clinical trials across various medication classes has provided new insights and knowledge. With the evolution of PH treatments reaching a new milestone, prompt and accurate diagnosis is increasingly critical. Developing early, effective management and treatment plans is essential to improve the long-term quality of life for PH patients.

show abstract

Section: Ertmentioning

confidence: 99%

Navigating the Evolving Landscape of Primary Hyperoxaluria: Traditional Management Defied by the Rise of Novel Molecular Drugs

Huang,

Zhu,

Zhou

et al. 2024

Biomolecules

View full text Add to dashboard Cite

show abstract

“…Another method of direct ERT involves the delivery of lipid nanoparticle-encapsulated mRNA. Successful delivery of AGT mRNA was demonstrated in Agxt -knockout mice, with a resultant 40% reduction in urinary oxalate 107 . Finally, direct ERT can also be performed through viral delivery of AGT complementary DNA (cDNA), which has been shown to significantly reduce urine oxalate by 2.7–3.6-fold ( P < 0.05) in Agxt -knockout mice in two studies 108 , 109 .…”

Section: Preclinical Strategies In Developmentmentioning

confidence: 99%

Perspectives in primary hyperoxaluria — historical, current and future clinical interventions

Shee

Stoller

2021

Nat Rev Urol

View full text Add to dashboard Cite

Primary hyperoxalurias are a devastating family of diseases leading to multisystem oxalate deposition, nephrolithiasis, nephrocalcinosis and end-stage renal disease. Traditional treatment paradigms are limited to conservative management, dialysis and combined transplantation of the kidney and liver, of which the liver is the primary source of oxalate production. However, transplantation is associated with many potential complications, including operative risks, graft rejection, post-transplant organ failure, as well as lifelong immunosuppressive medications and their adverse effects. New therapeutics being developed for primary hyperoxalurias take advantage of biochemical knowledge about oxalate synthesis and metabolism, and seek to specifically target these pathways with the goal of decreasing the accumulation and deposition of oxalate in the body.

show abstract

Disorders of Oxalate Metabolism

Höppe¹,

Beck²,

Martín-Higueras³

2022

Physician's Guide to the Diagnosis, Treatment, and Follow-Up of Inherited Metabolic Diseases

View full text Add to dashboard Cite

Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1

Cited by 13 publications

References 36 publications

Navigating the Evolving Landscape of Primary Hyperoxaluria: Traditional Management Defied by the Rise of Novel Molecular Drugs

Navigating the Evolving Landscape of Primary Hyperoxaluria: Traditional Management Defied by the Rise of Novel Molecular Drugs

Perspectives in primary hyperoxaluria — historical, current and future clinical interventions

Disorders of Oxalate Metabolism

Contact Info

Product

Resources

About