Abstract

The short film (15 mins) use animation, live action and interviews to introduce scientific principles, research and devselopment of major milestones in gene therapy over the last twenty years. We will discuss with the scientific team how to clarify what gene therapy is, and what scientific processes are involved, as well as the resulting societal issues. In order to show the detail of essential gene therapy processes such as base editing, the film focuses on three diseases of haemotapoetic cells, each of which have promising gene therapy treatments: Severe Combined Immonodeficiency Disease, Sickle Cell Disease and the use of Car-T cells to treat specific kinds of blood cancer. The film will be supplemented with a digital resource kit, the ‘toolkit’ which will give further textual and visual resources and references, allowing the film to focus on clarifying the underlying scientific processes as well as telling the story through patient’s experiences.
Original languageEnglish
Media of outputFilm
Size17 mins
Publication statusPublished - 2025

Keywords / Materials (for Non-textual outputs)

  • gene therapy
  • sickle-cell disease
  • gene editing
  • Severe Combined Immunodeficiency
  • CAR-T
  • lymphoid neoplasms
  • patient access to treatment

Type (for Non-textual outputs)

  • Film

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