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Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

Article Abstract:

The authors injected an animal-modeled version of hemophilia B, also known as factor IX deficiency, into mice and dogs. No toxic effects occurred in the animals and factor IX was produced in the mice for more than 17 months, with a smaller amount produced in the dogs for eight months or more.

Author: Kay, Mark A., Meuse, Leonard, Stafford, Darrel W., Bellinger, Dwight A., Read, Marjorie S., Brinkhous, Kenneth M., Nichols, Timothy C., Snyder, Richard O., Miao, Carol, Tubb, Julie, Donahue, Brian A., Lin, Hui-Feng, Patel, Salil, Thompson, Arthur R.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 1999

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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

Article Abstract:

The authors treated hemophilia B in five dogs using an adeno-virus vector for gene transfer. They transferred the missing factor IX and achiever long lasting results, with the disease staying in abeyance for more than 17 months, and with no or minimal adverse immune responses.

Author: High, Katherine A., Herzog, Roland W., Yang, Edmund Y., Couto, Linda B., Hagstrom, J. Nathan, Elwell, Dan, Fields, Paul A., Burton, Melissa, Bellinger, Dwight A., Read, Marjorie S., Brinkhous, Kenneth M., Podsakoff, Gregory M., Nichols, Timothy C., Kurtzman, Gary J.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 1999
Genetic engineering

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Coaxing coagulation with RNA and cell fragments

Article Abstract:

Two studies were conducted, which described findings that could lead to new approaches for the treatment of hemophilia. The study broadens the understanding of clot formation and also highlights another problem with this class of treatment strategies.

Author: High, Katherine A.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 2003
Science & research, Care and treatment, Hemophilia, RNA, Coagulation

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Subjects list: Health aspects, Research, Hemophilia B, Prothrombin complex concentrate, Factor IX (Blood coagulation)
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