Title : AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.

Pub. Date : 2020 Sep 11

PMID : 32775495






1 Functional Relationships(s)
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Compound Name
Protein Name
Organism
1 Deficient alpha-Gal A activity results in the progressive, systemic accumulation of its substrates, globotriaosylceramide (Gb3) and globotriaosylsphingosine (Lyso-Gb3), leading to renal, cardiac, and/or cerebrovascular disease and early demise. globotriaosylceramide alpha 1,4-galactosyltransferase (P blood group) Homo sapiens