https://fakinhibitors.com/envi....ronmentally-friendly
In Fabry mice, AAV8-FLT190 therapy resulted in approval of globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3) in plasma, urine, renal, and heart; electron microscopy analyses verified reductions in storage inclusion bodies in kidney and heart. In NHPs, α-Gal A expression was in keeping with the amount of hGLA mRNA in liver, with no FLT190-related toxicities or adverse events were seen. Taken together, these scientific studies indicate preclinical proof-of-concept of liver-
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