The First Journal on Human Gene Therapy Celebrates its 25th Anniversary

摘要: This story began in 1990 when a handful of scientists working the nascent field gene therapy came together with young, forward-thinking publishing company run by Mary Ann Liebert to launch provocative new journal. At time, science was thin but expectation its broad-based clinical success very thick. In inaugural issue Human Gene Therapy, then Editor-In-Chief French Anderson stated that “We plan for Therapy evolve this major frontier and document progress setbacks as we go.” (Anderson, 1990). The Journal indeed stayed true mission during rich storied history. played role translational behind first trials vivo ex therapy. The debate over these much about ethics public policy humans it science; served forum present complex issues. formative stages are chronicled almost verbatim early issues Journal. Human expanded exponential growth venue outstanding preclinical stakeholders went back bench after some clinic. As you know, community “stayed course.” With strong foundation technology, now seeing crescendo good news through successful trial results exciting directions. To keep step era, created two sibling journals: Methods (Wilson, 2011), provide technology will enable succeed, Clinical Development 2012), capture important research on regulatory sciences. Establishing journal 2013 brought us full circle days which also focused key albeit under different circumstances. No longer striving success; current efforts attempt leverage recent successes across broader array diseases facilitate access distribution patients. The incredible impact recognized 2009 National Libraries Association, identified “One 100 Most Influential Biomedical Life Science Journals Last Years.” documenting Glybera, commercially approved product West, illustrates how broad substantive has been. Glybera is an adeno-associated virus type 1–based European Medicines Agency 2012 use patients rare form high triglycerides due deficiency lipoprotein lipase. Virtually every milestone involved development published one journals, including proof-of-concept studies mouse feline models disease provided rationale clinic (Ross et al., 2004, 2006), investigational drug–enabling safety initial characterization potential human subjects (Rip 2005), molecular analyses phase 2 (Ferreira 2013), extensive review approval (Bryant 2013). We celebrate our silver anniversary several ways, beginning recognition pioneering scientists. A blue ribbon panel investigators convened identify 12 individuals who have made seminal contributions context career consistently contributed cell extended period time. committee, chaired Dr. Collins, defined contribution groundbreaking study or basic/technical advance substantially influenced direction and/or trajectory research. instances, Pioneer Award shared among whose work within programmatic area could not be fairly singled out. Each pioneer contribute perspective piece their future directions 2014 Therapy. composition Series Selection Committee awardees can found pages iv–v issue. This designed scientific developments well tradition discussing ethical addition number first-rate peer-reviewed articles, feature series articles from regarding Recombinant DNA Advisory (RAC), organization leadership governance United States. coincides deliberations Institute Medicine (IOM), committee evaluate RAC going forward; recommendations report entitled, “Oversight Review Transfer Protocols: Assessing Role Committee” Koehler al. (2014). We lead off coverage article Nelson Wivel—director NIH Office Activities 1986–1996—that chronicles history RAC. Testimonies participants IOM issue, commentary bioethicist Arthur Caplan concerning IOM's recommendations. The occasion provides opportunity reflect last 25 years. What strikes me resolve commitment believed would eventually succeed. Patients otherwise incurable benefiting therapy, cause celebration. However, only scratched surface field. need more improved technology. establish business support commercial so-called disruptive look forward day no described experimental emerging just standard medical care. journals continue evolution achieve goal.