It has been nearly 5 years since the last Human Molecular Genetics Gene Therapy compendium of reviews on the status of the field, and over this time we have seen a substantial progress, not only in numbers of trials but also in progression from early to late phase clinical studies for gene therapies in humans harboring inherited genetic disorders. The clinical studies being performed have moved from largely academic center investigator-initiated trials to now include the backing of new biotechnology companies focused on genetic medicines (either vector derived or nucleic acids and its derivatives) for disease treatment. And a most notable event over this period was the approval by the European Medicines Agency of the first gene therapy drug, for treatment of an inherited disorder (Glybera®).

We are witnessing the expected...

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