AAV5-miHTT gene therapy mediates sustained mutant huntingtin lowering in brain and cerebrospinal fluid of Huntington's disease minipigs up to 5 years

L. van der Bent, C. Brouwers,A. Stam, L. Spronck, J. Klima, J. Juhasova, B. Levinska,S. Juhas, J. Motlik,M. Evers, Z. Ellederova,A. Valles

MOVEMENT DISORDERS(2023)

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