High's gene therapy had cured hemophilia in mice and dogs and had caused rhesus monkeys to
express clotting factors.
Not exact matches
Today, many doctors consider anyone with a
clotting factor level well below normal to actually
express the disease.
Haemophiliacs and doctors have
expressed concern about the cost of a genetically engineered version of the vital blood -
clotting protein,
factor 8.
But rather than delivering the entire gene for the
clotting -
factor proteins to cells, as most gene therapies do, the researchers used the viruses to engineer immune - regulating B cells to
express a fragment of the
clotting factor fused to an immune molecule called an immunoglobulin.
In a chromosomal inversion, the order of the base pairs on the chromosome are reversed so the gene doesn't
express properly and the sufferer lacks the blood coagulation
factor VIII (F8) gene, which causes blood to
clot in healthy people.