Discovered in the mid-1960s, adenoassociated viruses (AAVs) have become the leading vector for gene therapy in recent years. In October 2012, the first European market authorization for a human genetherapy product was granted for UniQure’s Glybera (alipogene tiparvovec), which contains an AAV1 vector for treating patients who have lipoprotein lipase deficiency. (The product has been withdrawn from the market because of limited demand.) Both gene therapies currently approved in the United States — Luxturna (voretigene neparvovec) from Spark Therapeutics, approved in 2017, and Zolgensma (onasemnogene abeparvovec) from Novartis, approved in 2019 - are AAV-based gene therapies. Most ongoing gene-therapy pipelines remain focused on treating monogenic and rare human diseases. Some patients already have realized great benefits even as these programs advance our understanding and bring hope for those with complex diseases.
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