Enzyme Replacement Therapy
Enzyme Replacement Therapy - Related Videos
07:52
Detection of Antibodies That Neutralize the Cellular Uptake of Enzyme Replacement Therapies with a Cell-based Assay
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Cited by 4 •
2018
Here we present a cell-based flow cytometry method to detect neutralizing antibodies or other factors that interfere with the cellular uptake of enzyme replacement therapies in a human matrix, such as cerebral spinal fluid (CSF) or human serum.
01:30
Continuous Renal Replacement Therapy
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2025
Continuous Renal Replacement Therapy, also known as CRRT, is a procedural treatment for acute kidney injury (AKI) that gradually removes uremic toxins and fluids while maintaining acid-base balance and stabilizing electrolytes. It is particularly useful for hemodynamically unstable patients. Unlike intermittent hemodialysis, which is faster, CRRT provides a gentler approach over 24 hours, closely mimicking the function of natural kidneys. However, CRRT is not ideal for patients with...
01:26
Extracorporeal Removal of Drugs: Continuous Renal Replacement Therapy
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2025
Continuous Renal Replacement Therapy (CRRT) is an essential intervention for patients experiencing severe kidney dysfunction. This therapy offers a continuous mechanism for removing fluids and toxins from the bloodstream, leveraging the patient’s blood pressure to facilitate filtration through a specialized filter. This method contrasts with intermittent dialysis, providing a gentler and more consistent removal of waste products and excess fluid, which is particularly beneficial in critically...
09:34
Preparation of Plasma Membrane Vesicles from Bone Marrow Mesenchymal Stem Cells for Potential Cytoplasm Replacement Therapy
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Cited by 10 •
2017
Age-related diseases are associated with multiple defects in components of the cytoplasm. Here, we present a protocol to prepare plasma membrane vesicles from bone marrow mesenchymal stem cells. This technique could potentially be used as a means of cytoplasm replacement therapy to ameliorate or even reverse age-associated phenotypes.
02:34
Enzymes
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2020
Inside living organisms, enzymes act as catalysts for many biochemical reactions involved in cellular metabolism. The role of enzymes is to reduce the activation energies of biochemical reactions by forming complexes with its substrates. The lowering of activation energies favor an increase in the rates of biochemical reactions. Enzyme deficiencies can often translate into life-threatening diseases. For example, a genetic abnormality resulting in the deficiency of the enzyme G6PD...
FAQs
Cellular targeting determines whether the administered enzyme can act where substrate accumulates. After intravenous delivery, cells internalize the recombinant protein through receptor-mediated endocytosis, directing it into intracellular compartments such as lysosomes. This routing is important because restoring catalytic activity inside those compartments, rather than merely placing enzyme in circulation, supports substrate processing and cellular function.
Anti-drug antibodies can affect both the effectiveness and safety of enzyme replacement therapy. By reacting against the administered recombinant enzyme, they may interfere with treatment performance or contribute to unwanted responses. Their presence provides an immunological explanation for why the same replacement strategy may not produce identical outcomes across treated individuals when enzyme delivery or activity is affected.
An inherited enzyme deficiency can disrupt immune-cell metabolism, making its effects relevant to immunology as well as metabolic disease. ERT research therefore considers not only whether deficient catalytic activity is restored, but also how enzyme loss relates to immune-cell biology. This connection helps investigators study links between intracellular metabolism and cellular dysfunction in immune-related contexts.
A typical approach uses a recombinant enzyme prepared for intravenous administration. Once delivered, the protein must remain available for uptake by cells, enter through receptor-mediated endocytosis, and reach the relevant intracellular compartment. The intended outcome is restoration of deficient catalytic activity, followed by reduced harmful substrate accumulation and improved cellular function.
ERT is used primarily for inherited metabolic disorders, especially lysosomal storage disorders in which deficient enzyme activity leads to harmful substrate accumulation. By supplying functional enzyme, treatment aims to limit the cellular consequences of storage and reduce progressive organ damage. The approach is therefore most relevant when disease mechanisms are tied to a specific enzyme deficiency.
Evidence of benefit can be considered at several linked levels: deficient catalytic activity should be restored, harmful substrate accumulation should be reduced, and cellular function may improve. In disorders where storage damages organs, these cellular changes are relevant to the broader goal of limiting organ damage. This layered view distinguishes biochemical correction from downstream biological consequences in treatment and research.