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Comment
. 2016 Apr;24(4):666-7.
doi: 10.1038/mt.2016.50.

siRNA Therapeutics for Primary Hyperoxaluria: A Beginning

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Comment

siRNA Therapeutics for Primary Hyperoxaluria: A Beginning

Dawn S Milliner. Mol Ther. 2016 Apr.
No abstract available

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Figures

Figure 1
Figure 1
Kaplan–Meier plots of kidney survival among 168 patients with primary hyperoxaluria (PH) who did not have end-stage renal disease (ESRD) at diagnosis. Patients are shown by quartile of urine oxalate excretion (normal <0.45 mmol/1.73 m2/day). Patients in the highest quartile of urine oxalate excretion of ≥2.4 had the worst renal survival (42% at 20 years following diagnosis), compared with renal survival of 95% 20 years following diagnosis in those who had <1.6 mmol/1.73 m2/day. Reprinted from ref. .

Comment on

References

    1. Bobbin, ML and Rossi, JJ (2016). RNA interference (RNAi)-based therapeutics: delivering on the promise? Annu Rev Pharmacol Toxicol 56: 103–122. - PubMed
    1. Martin-Higueras, C, Luis-Lima, S and Salido, E (2016). Glycolate oxidase is a safe and efficient target for substrate reduction therapy in a mouse model of primary hyperoxaluria type I. Mol Ther 24: 719–725. - PMC - PubMed
    1. Dutta, C, Avitahl-Curtis, N, Pursell, N, Larsson Cohen, M, Holmes, B, Diwanji, R et al. (2016). Inhibition of glycolate oxidase with Dicer-substrate siRNA reduces calcium oxalate deposition in a mouse model of primary hyperoxaluria type I. Mol Ther 24: 770–778. - PMC - PubMed
    1. Coelho T, Adams, D, Silva, A, Lozeron, P, Hawkins, PN, Mant, T et al. (2013). Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 369: 819–829. - PubMed
    1. Cochat, P and Rumsby, G (2013). Primary hyperoxaluria. N Engl J Med 369: 649–658. - PubMed

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