uniQure Announces Publications on AAV Gene Therapy Approach to Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) in the Journal Molecular Therapy Nucleic Acids

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Feb 15, 2019
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Data Highlighted in Two Publications Demonstrate Early Preclinical Proof of Concept of AAV-based Gene Therapy to Silence Mutated C9orf72 Gene, A Frequent Cause of Familial ALS and FTD