A conditional counterselectable Piga knockout in mouse embryonic stem cells for advanced...

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Promise of gene therapy for congenital neurologic disease due to GPI deficiency

DV Babushok, DE Sabatino - Molecular Therapy Methods & Clinical …, 2024 - cell.com
A recent study by Murakami et al. 1 describes a novel genetic treatment for inherited
glycophosphatidylinositol (GPI) deficiency (IGD) using adeno-associated virus (AAV)-based …
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