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Literature summary for 3.1.6.13 extracted from

  • Polito, V.A.; Cosma, M.P.
    IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice (2009), Am. J. Hum. Genet., 85, 296-301.
    View publication on PubMedView publication on EuropePMC
Search for more literature for 3.1.6.13

Application

Application Comment Organism
medicine early treatment of mucopolysaccharidosis type II mice with one systemic injection of AAV2/5CMV-hIDS results in prolonged and high levels of circulating IDS that can efficiently and simultaneously rescue both visceral and central nervous system defects for up to 18 months after therapy Homo sapiens

Cloned(Commentary)

Cloned (Comment) Organism
AAV type 2/5 vector carrying human IDS cDNA under the control of the cytomegalovirus promoter (AAV2/5CMV-hIDS) administered in day 2 idsy/- mouse pups Homo sapiens

Organism

Organism UniProt Comment Textmining
Homo sapiens
-
-
-

Source Tissue

Source Tissue Comment Organism Textmining
blood
-
 Homo sapiens
-

Synonyms

Synonyms Comment Organism
IDS
-
 Homo sapiens
iduronate 2-sulfatase
-
 Homo sapiens

Expression

Organism Comment Expression
Homo sapiens no IDS expression in the brain samples of the control (wild-type and idsy/-) or treated (after 1 month or 18 months of AAV2/5CMV-hIDS therapy) mice down
Homo sapiens IDS expression in the liver and lungs of the control (wild-type and idsy/-) or treated (after 1 month or 18 months of AAV2/5CMV-hIDS therapy) mice up

General Information

General Information Comment Organism
physiological function in AAV2/5CMV-hIDS-injected mucopolysaccharidosis type II mice with Hunter syndrome, rescue of brain defects. The central nervous system correction arises from the crossing of the blood-brain barrier by the IDS enzyme Homo sapiens