Fabry Disease

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Genome-edited iPSCs show promise for treating Fabry Disease

4 July 2023 | By Izzy Wood (Drug Target Review)

Japanese researchers successfully engineered iPSCs to secrete a modified enzyme, mNAGA, and restored enzyme activity in vitro and in a mouse model, opening new avenues for regenerative medicine for conditions such as Fabry Disease.

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Fabry Disease

Genome-edited iPSCs show promise for treating Fabry Disease