Abstract
Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand. Genetic correction of the hemophilias represents a model system to develop a basic understanding of how gene therapy will be achieved. The goals for hemophilia gene transfer require the long-term therapeutic production of the coagulant protein without stimulating an immune response to the transgene product or the vector. Based on a scientific understanding of the molecular and cellular defects, leading to the bleeding phenotype, impressive strides have been made in the last 2 years.
Original language | English |
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Pages (from-to) | 999-1003 |
Number of pages | 5 |
Journal | Gene Therapy |
Volume | 10 |
Issue number | 12 |
DOIs | |
State | Published - Jun 2003 |
Keywords
- Gene repair
- Gene transfer
- Hemophilia
- Stem cells
- Viral vectors