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gene therapy for hemophilia B
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创建时间：
2017-11-21
相关数据集
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
Mice generated by disrupting the clotting factor IX gene exhibit severe bleeding disorder and closely resemble the phenotype seen in hemophilia B patients. Here we demonstrate that a single intraporta
PubMed Central1999-03-30 更新30
Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function
Mutations that result in amino acid changes can affect both pre-mRNA splicing and protein function. Understanding the combined effect is essential for correct diagnosis and for establishing the most a
NIAID Data Ecosystem20
Expression of human factor IX in mice after injection of genetically modified myoblasts.
Hemophilia B is an X chromosome-linked recessive bleeding disorder. To develop a somatic gene therapy for this disease, we have examined whether mouse skeletal myoblasts can serve as efficient vehicle
PubMed Central1992-04-15 更新20
Anatrichus pygmaeus Raw sequence reads
To prepare the manuscript "The complete mitochondrial genome of Antarichus pygmaeu lamb, 1918 (Diptera: Chloropidae)"
NIAID Data Ecosystem30
Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: potential for gene therapy of hemophilia B.
Hemophilia B (Christmas disease) is a chromosome X-linked blood clotting disorder which results when factor IX is deficient or functionally defective. The enzyme is synthesized in the liver, and the e
PubMed Central20
