RNA Interference Therapy for Machado-Joseph Disease: Long-Term Safety Profile of Lentiviral Vectors Encoding Short Hairpin RNAs Targeting Mutant Ataxin-3

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Authors: Nóbrega C, Codêsso JM, Mendonça L, Pereira de Almeida L.

Journal title: Human Gene Therapy

Journal publisher: Mary Ann Liebert Inc.

Published year: 2019

DOI identifier: 10.1089/hum.2018.157

ISSN: 1043-0342