Parvovirus-mediated gene transfer for the haemophilias
| Autor: | H. Chao, C. E. Walsh |
|---|---|
| Rok vydání: | 2002 |
| Předmět: |
viruses
Genetic enhancement Blotting Western Genetic Vectors Hemophilia A Haemophilia Virus Factor IX Immune system medicine Humans Vector (molecular biology) Serotyping Genetics (clinical) Factor VIII biology Parvovirus Gene Transfer Techniques Genetic Therapy Hematology General Medicine Dependovirus biology.organism_classification medicine.disease Virology Blot medicine.drug |
| Zdroj: | Haemophilia. 8:60-67 |
| ISSN: | 1365-2516 1351-8216 |
| Popis: | Gene therapy may revolutionize the treatment of haemophilia. Effective gene therapy requires sustained therapeutic levels of factors IX (FIX) and VIII. Adeno-associated virus (AAV) is a member of the parvovirus family, is a nonpathogenic virus with a broad host cell range, and does not provoke a significant immune response upon infection. These favourable characteristics make AAV a suitable gene transfer vector for factor deficient patients. A new understanding of AAV biology coupled with novel AAV vector designs suggest that the goal of effective gene transfer is within reach. We review here recent advances in AAV vectors used for gene transfer of the haemophilias. |
| Databáze: | OpenAIRE |
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