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Related Concept Videos

Lysosomal Hydrolases01:22

Lysosomal Hydrolases

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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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Proteoglycans01:05

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Glycans, a class of complex heterogeneous molecules, can be covalently attached to proteins to form glycosylated proteins that regulate various physiological and pathological processes. Glycosylated proteins or glycoproteins comprise N-linked and O-linked oligosaccharides. O-glycosylation is the most common type of protein glycosylation. Here, glycans attach to the oxygen atom of the hydroxyl groups of Serine or Threonine residues. O-linked glycosylation occurs later in protein processing,...
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Oligosaccharide Assembly01:24

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Protein glycosylation starts in the ER lumen and continues in the Golgi apparatus. Glycosyltransferases catalyze the addition of sugar molecules or glycosylation of proteins. Usually, these enzymes add sugars to the hydroxyl groups of selected serine or threonine residues to form O-linked glycans or the amino groups of asparagine residues to form N-linked glycans. Different positions on the same polypeptide chain can contain differently linked glycans.
Multiple sugar molecules that may or may...
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Related Experiment Video

Updated: Mar 16, 2026

Metabolic Glycoengineering of Sialic Acid Using N-acyl-modified Mannosamines
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Brain Targeting in MPS-IIIA.

Nicolina Cristina Sorrentino, Alessandro Fraldi

    Pediatric Endocrinology Reviews : PER
    |August 6, 2016
    PubMed
    Summary

    Mucopolysaccharidosis type IIIA (MPS-IIIA), a rare childhood neurodegenerative disorder, lacks a cure. Systemic therapies crossing the blood-brain barrier show promise for treating CNS pathology in MPS-IIIA mouse models.

    Area of Science:

    • Neuroscience
    • Metabolic Disorders
    • Genetics

    Background:

    • Mucopolysaccharidosis type IIIA (MPS-IIIA) is a childhood metabolic neuropathology due to sulfamidase deficiency, leading to glycosaminoglycan accumulation.
    • It is a common lysosomal storage disorder (LSD) with no current cure, where neurodegeneration is the primary pathology.
    • Effective treatment requires addressing central nervous system (CNS) lesions.

    Purpose of the Study:

    • To review current therapeutic strategies for MPS-IIIA, focusing on CNS involvement.
    • To highlight the success of systemic therapies that cross the blood-brain barrier (BBB) in preclinical models.
    • To discuss future clinical applications for MPS-IIIA and other neurological LSDs.

    Main Methods:

    • Review of preclinical and clinical studies on MPS-IIIA therapies.

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  • Focus on minimally invasive systemic approaches targeting CNS pathology.
  • Evaluation of therapeutic strategies in MPS-IIIA mouse and dog models.

    • Main Results:

    • Direct CNS administration routes are invasive and not clinically suitable.
    • Systemic therapies capable of crossing the BBB have shown success in treating CNS pathology and behavioral abnormalities in MPS-IIIA mouse models.
    • Preclinical models are crucial for developing and testing MPS-IIIA therapies.

    Conclusions:

    • Systemic therapeutic strategies crossing the BBB offer a promising avenue for treating MPS-IIIA CNS pathology.
    • Further clinical applications of these strategies are anticipated for MPS-IIIA patients.
    • Similar approaches may be beneficial for other LSDs with neurological involvement.