Modifying enzyme replacement therapy - A perspective.
Philipp SchaiblePublished in: Journal of cellular and molecular medicine (2022)
Several diseases are caused by the lack of functional proteins, including lysosomal storage diseases or haemophilia A and B. Patients suffering from one of these diseases are treated via enzyme replacement therapies to restore the missing protein. Although this treatment strategy prevents some disease symptoms, enzyme replacement therapies are very expensive and require very frequent infusions, which can cause infusion adverse reactions and massively impair the quality of life of the patients. This review proposes a technology to sustainably produce proteins within the patient to potentially make frequent protein-infusions redundant. This technology is based on blood circulating immune cells as producers of the needed therapeutic protein. To ensure a stable protein concentration over time the cells are equipped with a system, which induces cell proliferation when low therapeutic protein levels are detected and a system inhibiting cell proliferation when high therapeutic protein levels are detected.
Keyphrases
- cell proliferation
- end stage renal disease
- protein protein
- replacement therapy
- newly diagnosed
- chronic kidney disease
- ejection fraction
- binding protein
- peritoneal dialysis
- prognostic factors
- emergency department
- induced apoptosis
- small molecule
- cell cycle
- signaling pathway
- depressive symptoms
- smoking cessation
- combination therapy