Zobrazeno 1 - 10
of 14
pro vyhledávání: '"Julia Hettinger"'
Autor:
Garvin Warner, Wendell P Davis, Amy Chan, Jessica E. Sutherland, Jason A. Gilbert, Julia Hettinger
Publikováno v:
Nucleic Acid Therapeutics
Revusiran is a 1st-generation short interfering RNA targeting transthyretin conjugated to an N-acetylgalactosamine ligand to facilitate delivery to hepatocytes via uptake by the asialoglycoprotein receptors. Revusiran, in development for the treatmen
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::01e637ef54500d1abd7c76819946102d
http://europepmc.org/articles/PMC6987735
http://europepmc.org/articles/PMC6987735
Autor:
James Butler, Mark D. Fleming, Shannon Fishman, Paul J. Schmidt, Tim Racie, Julia Hettinger, Kaifeng Liu, Kevin Fitzgerald, Gary A. Visner
Publikováno v:
American Journal of Hematology. 93:745-750
Diminished β-globin synthesis in β-thalassemia is associated with ineffective erythropoiesis, leading to secondary iron overload caused by inappropriately low levels of hepcidin and to splenomegaly in the symptomatic thalassemias. Splenectomy is of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dad40cb9ca899f06fb3d0b4b78ef66fc
https://doi.org/10.1002/ajh.25079
https://doi.org/10.1002/ajh.25079
Autor:
Patrick Haslett, Timothy Racie, William Querbes, David Erbe, Abigail Liebow, Julia Hettinger, Brian Bettencourt, John Knight, Xingsheng Li, Ross P. Holmes, Kevin Fitzgerald, Nader Najafian
Publikováno v:
Journal of the American Society of Nephrology. 28:494-503
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 oxalate production resul
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bed87ff05ad92abfa79a372a3429b2ba
https://doi.org/10.1681/asn.2016030338
https://doi.org/10.1681/asn.2016030338
Autor:
Richard D Beckwitt, Nickolas Breen, William E. Barklow, Nathalia Vieira, Cesar Sanchez, Julia Hettinger, Jessica Barbagallo, Angelo Liquori
Publikováno v:
African Zoology. 51:77-82
Populations of Hippopotamus amphibius have declined throughout Africa in recent years, and are expected to decline further. An understanding of the population genetics of individual populations of hippos is necessary for effective management. To that
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::68df295a875de27f7870aebfb8bb6a9b
https://doi.org/10.1080/15627020.2016.1174076
https://doi.org/10.1080/15627020.2016.1174076
Autor:
Christopher A. Alabi, Tatiana Novobrantseva, Roman L. Bogorad, William Querbes, Weiheng Wang, Abigail K. R. Lytton-Jean, Daniel G. Anderson, Avi Schroeder, Akin Akinc, Arturo J. Vegas, Hao Yin, Carmen Barnes, Yi Chen, Delai Chen, Kevin Fitzgerald, J. Robert Dorkin, June Qin, Scott A Barros, Victor Koteliansky, Yizhou Dong, Robert Langer, Julia Hettinger, Sasilada Sirirungruang, Gaurav Sahay, Kevin T. Love, Daniel J. Siegwart, Mary Carioto, Varun Kumar, Yunlong Zhang, Karsten Olejnik
Publikováno v:
Proceedings of the National Academy of Sciences. 111:3955-3960
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 therapeutic index. Lead material cKK-E12 showed
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::40bcc7ad1c737c8c82f110498a4ee39a
https://doi.org/10.1073/pnas.1322937111
https://doi.org/10.1073/pnas.1322937111
Autor:
Christian Wolfrum, Nikolay V. Dokholyan, Gang Wang, Valerie A. Clausen, Julia Hettinger, Svetlana Shulga Morskaya, Qianfan Wang, Joshua Brodsky, Verbena Kosovrasti, Xuemei Zhang, Dinah W.Y. Sah, Mike Meys, Kallanthottathil G. Rajeev, Yupang Fang, Muthiah Manoharan, Jennifer Sharman, Martin Maier, Feng Ding, Mariano Severgnini, Victor Kotelianski, Lubomir Tchangov, Renta Hutabarat, Chang Geng Peng, Robert Li, Tim Racie, Lubomir Nechev, James Butler, Tomoko Nakayama, Scott A Barros, Alfica Sehgal, Markus Stoffel
Publikováno v:
Molecular Therapy, 20 (8)
Molecular Therapy
MOLECULAR THERAPY
Molecular Therapy
MOLECULAR THERAPY
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 the corresponding pathogenic protein. The major challenge
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2b95aa66e5836fecaa23e9693ac24ddd
https://doi.org/10.1038/mt.2012.33
https://doi.org/10.1038/mt.2012.33
Autor:
Amy Simon, Brian C. Cooley, Akin Akinc, Julia Hettinger, Renta Hutabarat, Yongfeng Jiang, Rachel Meyers, June Qin, Mary Carioto, Martin Maier, Klaus Charisse, Harsha K. Prabhala, Husain Attarwala, Rodney M. Camire, Lubo Nechev, Pachamuthu Kandasamy, Lacramioara Ivanciu, Alexander V Kel'in, Muthiah Manoharan, Kallanthottathil G. Rajeev, Scott A Barros, Jayaprakash K. Nair, Don Foster, Satya Kuchimanchi, Yesim Dargaud, Xuemei Zhang, Benny Sørensen, Tim Racie, Stuart Milstein, Alfica Sehgal, Claude Negrier, Josh Brodsky
Publikováno v:
Nature medicine. 21(5)
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, especially in patients with inhibito
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e4aa616e2a51cb586e3970c1386fe429
https://pubmed.ncbi.nlm.nih.gov/25849132
https://pubmed.ncbi.nlm.nih.gov/25849132
Autor:
Paul J. Schmidt, Julia Hettinger, Mark D. Fleming, David Bumcrot, Brian Bettencourt, Tim Racie, Stuart Milstein, Anoop K. Sendamarai, Ivanka Toudjarska
Publikováno v:
Blood. 121(7)
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 congenital anemia caused by part
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::533cec9e229c47c50fee197706137597
https://pubmed.ncbi.nlm.nih.gov/23411732
https://pubmed.ncbi.nlm.nih.gov/23411732
Autor:
Yongfeng Jiang, June Qin, Harsha K. Prabhala, Akin Akinc, Julia Hettinger, Mary Carioto, Scott A Barros, Alfica Sehgal
Publikováno v:
Blood. 122:3585-3585
ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), is currently being developed for the treatment of hemophilia and rare bleeding disorders. It has previously been demonstrated that once weekly dosing of ALN-AT3 resu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::466e3397555f263bd0307cca4c1e8b39
https://doi.org/10.1182/blood.v122.21.3585.3585
https://doi.org/10.1182/blood.v122.21.3585.3585
Autor:
Julia Hettinger, Tim Racie, James Butler, Brian Bettencourt, Klaus Charisse, Kevin Fitzgerald, Shannon Fishman
Publikováno v:
Blood. 122:2260-2260
The b-Thalassemias are a group of hereditary blood disorders resulting from insufficient beta globin production, ultimately giving rise to the signature clinical sequelae associated with β-Thalassemia which includes anemia, ineffective erythropoiesi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::4af7cec3ad251b9c7d069bccc14392ac
https://doi.org/10.1182/blood.v122.21.2260.2260
https://doi.org/10.1182/blood.v122.21.2260.2260