AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Elisabeth A Spronck, Cynthia Brouwers, Astrid Vallès, Martin de Haan, Harald Petry, S J van Deventer, Pavlina Konstantinova, Melvin M. Evers

Molecular Therapy — Methods & Clinical Development · 2019 · 85 citations · 26 references

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