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- Molecular_chaperone_therapy abstract "Molecular chaperone therapy is one of the latest pharmacological approaches to lysosomal storage diseases. It fixes defective protein as an alternative to Stop codon suppression treatment. These chaperones are minute molecules that can enter the central nervous system ( via Blood Brain Barrier). Once in the CNS, they attach to the enzyme (inactive form) and fix it so that it takes the correct functional shape. Limitation of the therapy is that it only works with certain mutations : Gaucher's disease, Fabry disease, Pompe disease and Late-onset Tay-Sachs disease. Lack of bio-marker and the potential to reduce the enzyme function if given incorrect dose are challenging aspects of current clinical trials.".
- Molecular_chaperone_therapy wikiPageID "33442595".
- Molecular_chaperone_therapy wikiPageLength "900".
- Molecular_chaperone_therapy wikiPageOutDegree "10".
- Molecular_chaperone_therapy wikiPageRevisionID "641404502".
- Molecular_chaperone_therapy wikiPageWikiLink Blood-brain_barrier.
- Molecular_chaperone_therapy wikiPageWikiLink Blood–brain_barrier.
- Molecular_chaperone_therapy wikiPageWikiLink Category:Lysosomal_storage_diseases.
- Molecular_chaperone_therapy wikiPageWikiLink Central_nervous_system.
- Molecular_chaperone_therapy wikiPageWikiLink Clinical_trial.
- Molecular_chaperone_therapy wikiPageWikiLink Fabry_disease.
- Molecular_chaperone_therapy wikiPageWikiLink Gauchers_disease.
- Molecular_chaperone_therapy wikiPageWikiLink Glycogen_storage_disease_type_II.
- Molecular_chaperone_therapy wikiPageWikiLink Lysosomal_storage_disease.
- Molecular_chaperone_therapy wikiPageWikiLink Pompe_disease.
- Molecular_chaperone_therapy wikiPageWikiLink Stop_codon.
- Molecular_chaperone_therapy wikiPageWikiLink Tay-Sachs_disease.
- Molecular_chaperone_therapy wikiPageWikiLink Tay–Sachs_disease.
- Molecular_chaperone_therapy wikiPageWikiLinkText "molecular chaperone therapy".
- Molecular_chaperone_therapy hasPhotoCollection Molecular_chaperone_therapy.
- Molecular_chaperone_therapy wikiPageUsesTemplate Template:Multiple_issues.
- Molecular_chaperone_therapy subject Category:Lysosomal_storage_diseases.
- Molecular_chaperone_therapy type Article.
- Molecular_chaperone_therapy type Article.
- Molecular_chaperone_therapy type Error.
- Molecular_chaperone_therapy comment "Molecular chaperone therapy is one of the latest pharmacological approaches to lysosomal storage diseases. It fixes defective protein as an alternative to Stop codon suppression treatment. These chaperones are minute molecules that can enter the central nervous system ( via Blood Brain Barrier). Once in the CNS, they attach to the enzyme (inactive form) and fix it so that it takes the correct functional shape.".
- Molecular_chaperone_therapy label "Molecular chaperone therapy".
- Molecular_chaperone_therapy sameAs m.0h965nb.
- Molecular_chaperone_therapy sameAs Q6895995.
- Molecular_chaperone_therapy sameAs Q6895995.
- Molecular_chaperone_therapy wasDerivedFrom Molecular_chaperone_therapy?oldid=641404502.
- Molecular_chaperone_therapy isPrimaryTopicOf Molecular_chaperone_therapy.