Literature summary for 3.1.6.8 extracted from

Capotondo, A.; Cesani, M.; Pepe, S.; Fasano, S.; Gregori, S.; Tononi, L.; Venneri, M.A.; Brambilla, R.; Quattrini, A.; Ballabio, A.; Cosma, M.P.; Naldini, L.; Biffi, A.
Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy (2007), Hum. Gene Ther., 18, 821-836.

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Application

Application	Comment	Organism
medicine	therapeutic levels of ARSA overexpression can be safetly achieved. ARSA-transduced cells efficiently repopulate the hematopoietic organs of RAG2-/- gamma-chain-/- mice. ARSA overexpression does not impair clonogenic capacity and multilineage differentiation of human HSPC cells, activation of other sulfatases and is thus unlikely to trigger metabolic imbalances. Moreover, ARSA overexpression does not impair immune functions, behavior and learning abilities	Homo sapiens

Cloned(Commentary)

Cloned (Comment)	Organism
HSPC cells transduced with increasing doses of lentiviral vectors expressing ARSA under the control of the human PGK promoter (PGK.ARSA.LV). Transgenic mice generated with PGK.ARSA.LV overexpressing the ARSA enzyme	Homo sapiens

Organism

Organism	UniProt	Comment	Textmining
Homo sapiens	-	-	-

Substrates and Products (Substrate)

Substrates	Comment Substrates	Organism	Products	Comment (Products)	Rev.	Reac.
N-lissamine rhodaminyl-(12-aminododecanoyl)cerebroside 3-sulfate + H2O	-	Homo sapiens	?	-	?
p-nitrocatechol sulfate + H2O	-	Homo sapiens	p-nitrocatechol + sulfate	-	?

Synonyms

Synonyms	Comment	Organism
ARSA	-	Homo sapiens
arylsulfatase A	-	Homo sapiens

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Release 2023.1 (January 2023)