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Progress and challenges in viral vector manufacturing

30 Oct 2015

Abstract excerpt

Promising results in several clinical studies have emphasized the potential of gene therapy to address important medical needs and initiated a surge of investments in drug development and commercialization. This enthusiasm is driven by positive data in clinical trials including gene replacement for Hemophilia B, X-linked Severe Combined Immunodeficiency, Leber's Congenital Amaurosis Type 2 and in cancer...

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PMID: 26519140
DOI: 10.1093/hmg/ddv451

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