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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
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Lysosomes are membrane-enclosed spherical sacs derived from the Golgi apparatus. The most important function of the lysosome is degrading macromolecules and biological polymers that are released during membrane trafficking events such as the secretory, endocytic, autophagic, and phagocytic pathways. The degradation is carried out by several hydrolytic enzymes active in an acidic environment of the lysosomal lumen. These acid hydrolases are involved in cellular processes such as cell signaling,...
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Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...
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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Enzyme replacement for lysosomal diseases.

Roscoe O Brady1

  • 1Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892-1260, USA. bradyr@ninds.nih.gov

Annual Review of Medicine
|January 18, 2006
PubMed
Summary
This summary is machine-generated.

Enzyme replacement therapy (ERT) has become a highly effective treatment for Gaucher disease, a prevalent metabolic disorder. This success has paved the way for ERT in other lysosomal storage disorders, with ongoing advancements in the field.

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Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • Sphingolipid storage disorders stem from enzymatic defects identified in the 1960s.
  • Early therapeutic considerations focused on enzyme supplementation or replacement strategies.
  • Significant research and novel protein targeting were necessary to develop effective treatments.

Purpose of the Study:

  • To review the current status of enzyme replacement therapy (ERT) for lysosomal storage disorders.
  • To discuss anticipated future developments in ERT for these conditions.

Main Methods:

  • Review of historical research and clinical data on ERT.
  • Analysis of therapeutic outcomes in Gaucher disease.
  • Examination of the extension of ERT to other lysosomal storage disorders.

Main Results:

  • Enzyme replacement therapy (ERT) demonstrated extraordinary effectiveness in Gaucher disease, a common metabolic storage disorder.
  • The success in Gaucher disease facilitated the application of ERT to other lysosomal storage disorders.
  • Current ERT approaches represent a significant advancement in managing these genetic conditions.

Conclusions:

  • Enzyme replacement therapy is a proven and effective treatment for specific lysosomal storage disorders.
  • Continued research promises further advancements and broader applications of ERT.
  • The field is evolving, with ongoing developments expected to improve patient outcomes.