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Administration of an adeno-associated viral vector expressing interferon-beta in patients with inflammatory hand arthritis, results of a phase I/II study
Administration of an adeno-associated viral vector expressing interferon-β in patients with inflammatory hand arthritis, results of a phase I/II study
Intrastriatal administration of AAV5-miHTT in non-human primates and rats is well tolerated and results in miHTT transgene expression in key areas of Huntington disease pathology
MicroRNA-based gene therapy for Huntington's disease : Silencing the villain