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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1964 1
1974 1
1982 2
1984 2
1987 1
1988 2
1989 5
1990 3
1991 5
1992 2
1993 5
1994 10
1995 10
1996 12
1997 8
1998 14
1999 6
2000 4
2001 5
2002 6
2003 7
2004 10
2005 12
2006 8
2007 1
2008 4
2009 8
2010 9
2011 4
2012 7
2013 17
2014 7
2015 14
2016 11
2017 8
2018 13
2019 11
2020 10
2021 13
2022 18
2023 21
2024 11

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296 results

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Page 1
Chemistry, structure and function of approved oligonucleotide therapeutics.
Egli M, Manoharan M. Egli M, et al. Nucleic Acids Res. 2023 Apr 11;51(6):2529-2573. doi: 10.1093/nar/gkad067. Nucleic Acids Res. 2023. PMID: 36881759 Free PMC article. Review.
Among the diseases targeted by this new class of drugs are homozygous familial hypercholesterolemia, spinal muscular atrophy, Duchenne muscular dystrophy, hereditary transthyretin-mediated amyloidosis, familial chylomicronemia syndrome, acute hepatic porphyria, and primary
Among the diseases targeted by this new class of drugs are homozygous familial hypercholesterolemia, spinal muscular atrophy, Duchenne muscu …
Therapeutic siRNA: State-of-the-Art and Future Perspectives.
Friedrich M, Aigner A. Friedrich M, et al. BioDrugs. 2022 Sep;36(5):549-571. doi: 10.1007/s40259-022-00549-3. Epub 2022 Aug 23. BioDrugs. 2022. PMID: 35997897 Free PMC article. Review.
These are mainly based on an siRNA conjugation with a targeting moiety for liver hepatocytes, N-acetylgalactosamine, and cover the treatment of acute hepatic porphyria, transthyretin-mediated amyloidosis, hypercholesterolemia, and primary hyperoxaluria type 1. Still …
These are mainly based on an siRNA conjugation with a targeting moiety for liver hepatocytes, N-acetylgalactosamine, and cover the treatment …
Oxalate homeostasis.
Ermer T, Nazzal L, Tio MC, Waikar S, Aronson PS, Knauf F. Ermer T, et al. Nat Rev Nephrol. 2023 Feb;19(2):123-138. doi: 10.1038/s41581-022-00643-3. Epub 2022 Nov 3. Nat Rev Nephrol. 2023. PMID: 36329260 Free PMC article. Review.
Novel studies have shed light on the essential roles of metabolic pathways, the microbiome, epithelial oxalate transporters, and adequate oxalate excretion to maintain oxalate homeostasis. In patients with primary or secondary hyperoxaluria, nephrolithiasis, acute o …
Novel studies have shed light on the essential roles of metabolic pathways, the microbiome, epithelial oxalate transporters, and adequate ox …
Pathophysiology and Management of Hyperoxaluria and Oxalate Nephropathy: A Review.
Demoulin N, Aydin S, Gillion V, Morelle J, Jadoul M. Demoulin N, et al. Am J Kidney Dis. 2022 May;79(5):717-727. doi: 10.1053/j.ajkd.2021.07.018. Epub 2021 Sep 9. Am J Kidney Dis. 2022. PMID: 34508834 Review.
Hyperoxaluria results from either inherited disorders of glyoxylate metabolism leading to hepatic oxalate overproduction (primary hyperoxaluria), or increased intestinal oxalate absorption (secondary hyperoxaluria). ...Fortunately, novel promising targ
Hyperoxaluria results from either inherited disorders of glyoxylate metabolism leading to hepatic oxalate overproduction (primary
Primary hyperoxaluria.
Cochat P, Rumsby G. Cochat P, et al. N Engl J Med. 2013 Aug 15;369(7):649-58. doi: 10.1056/NEJMra1301564. N Engl J Med. 2013. PMID: 23944302 Review. No abstract available.
Clinical practice recommendations for primary hyperoxaluria: an expert consensus statement from ERKNet and OxalEurope.
Groothoff JW, Metry E, Deesker L, Garrelfs S, Acquaviva C, Almardini R, Beck BB, Boyer O, Cerkauskiene R, Ferraro PM, Groen LA, Gupta A, Knebelmann B, Mandrile G, Moochhala SS, Prytula A, Putnik J, Rumsby G, Soliman NA, Somani B, Bacchetta J. Groothoff JW, et al. Nat Rev Nephrol. 2023 Mar;19(3):194-211. doi: 10.1038/s41581-022-00661-1. Epub 2023 Jan 5. Nat Rev Nephrol. 2023. PMID: 36604599 Review.
Primary hyperoxaluria (PH) is an inherited disorder that results from the overproduction of endogenous oxalate, leading to recurrent kidney stones, nephrocalcinosis and eventually kidney failure; the subsequent storage of oxalate can cause life-threatening systemic
Primary hyperoxaluria (PH) is an inherited disorder that results from the overproduction of endogenous oxalate, leading to rec
Genetics of kidney stone disease.
Howles SA, Thakker RV. Howles SA, et al. Nat Rev Urol. 2020 Jul;17(7):407-421. doi: 10.1038/s41585-020-0332-x. Epub 2020 Jun 12. Nat Rev Urol. 2020. PMID: 32533118 Review.
Studies suggest that the prevalence of monogenic kidney stone disorders, including renal tubular acidosis with deafness, Bartter syndrome, primary hyperoxaluria and cystinuria, in patients attending kidney stone clinics is 15%. ...
Studies suggest that the prevalence of monogenic kidney stone disorders, including renal tubular acidosis with deafness, Bartter syndrome, …
Haemolytic uraemic syndrome.
Michael M, Bagga A, Sartain SE, Smith RJH. Michael M, et al. Lancet. 2022 Nov 12;400(10364):1722-1740. doi: 10.1016/S0140-6736(22)01202-8. Epub 2022 Oct 19. Lancet. 2022. PMID: 36272423 Review.
Obesity and kidney stone disease: a systematic review.
Carbone A, Al Salhi Y, Tasca A, Palleschi G, Fuschi A, De Nunzio C, Bozzini G, Mazzaferro S, Pastore AL. Carbone A, et al. Minerva Urol Nefrol. 2018 Aug;70(4):393-400. doi: 10.23736/S0393-2249.18.03113-2. Epub 2018 May 31. Minerva Urol Nefrol. 2018. PMID: 29856171 Free article. Review.
Contemporary, bariatric surgery has been shown to be associated with hyperoxaluria and oxalate nephropathy. Certainly, the many health risks of obesity, including nephrolithiasis, will add more burden on urologists and nephrologists. CONCLUSIONS: Obesity related nephrolith …
Contemporary, bariatric surgery has been shown to be associated with hyperoxaluria and oxalate nephropathy. Certainly, the many healt …
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
Conjugation of oligonucleotide therapeutics, including small interfering RNAs (siRNAs) or antisense oligonucleotides, to N-acetylgalactosamine (GalNAc) ligands has become the primary strategy for hepatocyte-targeted delivery, and with the recent approvals of GIVLAARI (givo …
Conjugation of oligonucleotide therapeutics, including small interfering RNAs (siRNAs) or antisense oligonucleotides, to N-acetylgalactosami …
296 results