Therapeutic approaches to genetic disorders

ABSTRACT

Although prevention is the ideal goal for genetic disorders, various types of therapeutic management are available. Such management approaches depend on the nature of the defect, how well it is understood at the genetic and biochemical levels and the practical feasibility of correction. In some conditions certain management is now tailored to the specific genotype. The patient being treated may be the fetus, the infant, the child or the adult. Treatment methods used in genetic disorders may involve surgical, cognitive/behavioral, pharmacologic, dietary, envairomental avoidance, transfusion, plasma exchange, enzyme, behavioral, cell, or gene therapy. Some have been developed on the basis of knowledge of the defect in the gene and its product, whereas others are empirical or aimed at controlling or mediating signs and symptoms without care