Sentences with phrase «defective gene for the enzyme»

Individuals with GSDIa have a defective gene for the enzyme glucose -6-phosphatase, resulting in the inability to regulate blood sugar (glucose).

DTX 401 is designed to address the defective gene for the enzyme glucose -6-phosphatase-α, a defect that results in the inability to regulate blood sugar (glucose).

This is clear from the fact that people with a defective gene for the LDL - receptor get heart disease very early — in infancy in the most extreme case — and people with a defective gene for an enzyme that degrades the LDL - receptor have virtual immunity to heart disease.

Not exact matches

Woo's technique may one day be used to treat the metabolic disorder phenylketonuria (PKU), in which the gene for the hepatic enzyme known as phenylalanine hydroxylase (PAH) is defective.

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).

Our lead candidate, LYS - GM101, has been designed to replace this defective gene in the cells of GM1 patients, in order to allow for production of the functional lysosomal acid beta - galactosidase (bgal) enzyme.