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  • Collection |

    See what scientists world-wide have been citing, reading and sharing. In this Web Collection we highlight a selection of articles from 2017 and 2018 which top the list of the journal's most cited, most read and most shared (including press coverage and Twitter). They showcase the breadth of scope and coverage that Gene Therapy consistently delivers to its readers.

  • Special |

    The Spinraza and Advanced Therapies: a stakeholder special issue of Gene Therapy covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected, experts in the advanced therapies field, SMA scientists and clinicians, regulators, ethics and pricing/IP experts, and community stakeholders.

  • Focus |

    The science of gene therapy is enjoying a renaissance after ups and downs over the past 25 years. The heart of this field still largely relies on viral vector gene transfer systems that have evolved considerably. There is now a wealth of clinical experience with numerous vector types that have been applied primarily to treatment of cancer and rare metabolic diseases. Viral vectors will likely achieve considerable prominence in the delivery of gene editing tools, providing an important aspect of gene therapy strategies going forward. Still more advanced platforms and vehicle delivery systems are required to enable appropriate level and duration of gene expression in targeted cell types. Recent research into the field of vector technologies are highlighted in publications in the journal Gene Therapy.