Julia Hettinger
Overview
Explore the profile of Julia Hettinger including associated specialties, affiliations and a list of published articles.
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Articles
6
Citations
793
Followers
0
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Recent Articles
1.
Liebow A, Li X, Racie T, Hettinger J, Bettencourt B, Najafian N, et al.
J Am Soc Nephrol
. 2016 Jul;
28(2):494-503.
PMID: 27432743
Primary hyperoxaluria type 1 (PH1), an inherited rare disease of glyoxylate metabolism, arises from mutations in the enzyme alanine-glyoxylate aminotransferase. The resulting deficiency in this enzyme leads to abnormally high...
2.
Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, et al.
Nat Med
. 2015 Apr;
21(5):492-7.
PMID: 25849132
Hemophilia A and B are inherited bleeding disorders characterized by deficiencies in procoagulant factor VIII (FVIII) or factor IX (FIX), respectively. There remains a substantial unmet medical need in hemophilia,...
3.
Dong Y, Love K, Dorkin J, Sirirungruang S, Zhang Y, Chen D, et al.
Proc Natl Acad Sci U S A
. 2014 Feb;
111(11):3955-60.
PMID: 24516150
siRNA therapeutics have promise for the treatment of a wide range of genetic disorders. Motivated by lipoproteins, we report lipopeptide nanoparticles as potent and selective siRNA carriers with a wide...
4.
Maier M, Jayaraman M, Matsuda S, Liu J, Barros S, Querbes W, et al.
Mol Ther
. 2013 Jun;
21(8):1570-8.
PMID: 23799535
In recent years, RNA interference (RNAi) therapeutics, most notably with lipid nanoparticle-based delivery systems, have advanced into human clinical trials. The results from these early clinical trials suggest that lipid...
5.
Schmidt P, Toudjarska I, Sendamarai A, Racie T, Milstein S, Bettencourt B, et al.
Blood
. 2012 Dec;
121(7):1200-8.
PMID: 23223430
Mutations in HFE lead to hereditary hemochromatosis (HH) because of inappropriately high iron uptake from the diet resulting from decreased hepatic expression of the iron-regulatory hormone hepcidin. -thalassemia is a...
6.
Nakayama T, Butler J, Sehgal A, Severgnini M, Racie T, Sharman J, et al.
Mol Ther
. 2012 Aug;
20(8):1582-9.
PMID: 22850721
Therapeutics based on RNA interference (RNAi) have emerged as a potential new class of drugs for treating human disease by silencing the target messenger RNA (mRNA), thereby reducing levels of...