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AAV-based gene therapy ameliorated CNS-specific GPI defect ..:

Murakami, Yoshiko ; Umeshita, Saori ; Imanishi, Kae...
Molecular Therapy - Methods & Clinical Development.  32 (2024)  1 - p. 101176 , 2024
Link: https://doi.org/10.1016/j.omtm.2023.101176