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Cell Press, Molecular Therapy, 9(26), p. 2163-2177, 2018

DOI: 10.1016/j.ymthe.2018.06.021

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AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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