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In vivo targeting of a variant causing vanishing white matt..:

Hillen, Anne E.J. ; Hruzova, Martina ; Rothgangl, Tanja...
Molecular Therapy - Methods & Clinical Development.  25 (2022)  - p. 17-25 , 2022
Link: https://doi.org/10.1016/j.omtm.2022.02.006