ASC - Gene Replacement Therapy

Genetic diseases are caused by defective genes. Gene replacement therapy aims to treat such diseases by introducing a corrective gene into a patient’s body; the corrective gene can be a normal copy of the defective gene, or a copy that is engineered to provide enhanced biological activity; our focus for gene replacement therapy starts by identifying gene variants with superior therapeutic potency and to achieve optimal and sustainable production of the functional protein once administered to the patient.