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Perspectives on cell and gene therapies

We summarise a recent systematic review of the current evidence on patient and public knowledge and perspectives of cell and gene therapies. Understanding these perspectives will help inform future research, education and awareness raising activities.

Cell and gene therapies

Over the last decade, there have been great advancements in cell, gene and tissue-engineered therapies. Several engineered therapies have been developed to treat various cancers, inherited diseases and chronic conditions. These therapies are approved and regulated in the US by the Center for Biologics Evaluation and Research and in Europe by the European Medicines Agency (EMA).

The delivery of these therapies presents logistical and delivery challenges for healthcare systems globally. Alongside this, there are complex social, ethical, health and economic issues that need to be considered. Patient engagement, awareness and understanding will be critical to ensure public contribution in policy debates and to enhance their ability to make informed decisions.

Perspectives on cell and gene therapies

In a study, published in Nature Communications, researchers used systematic approaches to summarise and discuss the current evidence on patient and public opinions on cell and gene therapies. They also explored the potential influences on their views and highlighted future priority areas for research. Specifically, they screened 10,735 titles and abstracts and evaluated the full texts of 151 publications. The final selection was 35 publications.

They identified four themes from the narrative synthesis of the study findings:

  • Knowledge and understanding of cell and gene therapies: The studies reported varying levels of patient knowledge and understanding of cell and gene therapies. In addition, just like patients, the public did not perceive themselves well informed on the subject and desired more information.
  • Acceptance of cell and gene therapies: Acceptance varied among patients but generally increased after the provision of information. Studies involving the public generally reported support for cell and gene therapy research. There was low public acceptance for these therapies for non-therapeutic use. Concerns included threat to genetic diversity, government interference with parent’s right to choose and costs. Reasons for support included increasing lifespan, improving intelligence and improving health and fitness.
  • Understanding of risk and benefits of therapy: A number of studies found unrealistic expectations around timescales and the benefits of these therapies. Some individuals expressed concerns about the benefit/risk balance. In particular, parents of younger children. Nonetheless, these parents’ tolerance for risk increased as their children grew older or health deteriorated.
  • Information needs and current sources of information: Patients frequently indicated a desire for more information regardless of their gender, age or educational status. Researchers identified television as the major source of information. However, it was reported that patients perceived physicians as the most reliable source of information.


Cell and gene therapies have the potential to change the treatment of diseases. Nonetheless, significant risks to patients and complex challenges translating these into clinical applications still exist. Patient and public perception are critical in the development of therapies and their subsequent uptake. Working with patients and the public to design further research and or educational resources is critical to address current issues.

Image credit: By pch.vector –

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