Sentences with phrase «hemophilia produced»

Not exact matches

For nearly two decades, gene therapy researchers have focused on delivering a gene carrying the code to produce the deficient clotting factor in hemophilia.

Scottish researchers took a similar approach to clone sheep that will produce in their milk human factor IX protein, which is used to treat hemophilia.

To verify that the process worked, the endothelial cells with the inversion - corrected genes were transplanted into F8 deficient mice (mice with hemophilia A) and the mice started producing the F8 clotting factor on their own, which essentially cured them of hemophilia A.

Other genetic diseases include Tay - Sachs disease (damage to the gene for the enzyme hexosaminidase A leads to an accumulation of a chemical in the brain that destroys it), sickle cell anemia (improper coding of the gene that produces hemoglobin), hemophilia (lack of a gene for a blood - clotting factor) and muscular dystrophy (caused by a defective gene on the X chromosome).

Years after receiving a single dose of gene therapy, patients on the hemophilia B trial continued to produce their own clotting factor from the normal transferred gene with minimal side effects.

The resultant clones with functional FIX can then be screened for integration of the viral backbone in safe harbor regions and for its robust expression, subsequent to which they will be differentiated in vitro to the hepatic lineage, with a view to producing a cell - based therapy for hemophilia.