A novel AAV-based miQURE gene therapy for SCA3

Toonen, LJA; Nobre, R; Haas, E; Hubener-Schmid, J; Martier, R; Valles, A; Higgens, JJ; van Deventer, S; Nguyen, HP; de Almeida, LP; Konstantinova, P; Evers, MM

HUMAN GENE THERAPY, 2019; 30 (11): A91

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