Recombinant protein is a manipulated form of protein generated using different approaches in order to produce large quantities of molecules to utilize as treatment option for specific diseases. Recombinant technology is a process based on the introduction of defined gene sequences in specialized vehicles known as vectors. These vectors are then introduced in expression systems to allow high quantity of recombinant protein production.
Enzyme replacement therapy (ERT) is a therapeutic approach through which a defined enzymatic protein is introduced in patients that have a deficit for a specific enzymatic activity.
ERT has been a revolution for some diseases treatment moving the treatment of these disorders from symptomatic management to therapeutic interventions that address the underlying metabolic defect. ERT does not represent a cure for these enzymatic disorders; however it can significantly modify and in some cases eliminate the signs and symptoms of the disease.