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AAV9-DOK7 gene therapy reduces disease severity in Smn SMA ..:

Kaifer, Kevin A. ; Villalón, Eric ; Smith, Caley E....
Biochemical and Biophysical Research Communications.  530 (2020)  1 - p. 107-114 , 2020
Link: https://doi.org/10.1016/j.bbrc.2020.07.031