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Parental perspectives on gene therapy for children with haemophilia: The Exigency study
Kate Khair1, Lisa Steadman1, Steve Chaplin1
1Haemnet, London, UK.
Insights
Parents of children with haemophilia are open to gene therapy, but require clear, factual information. A community-wide strategy, delivered by trusted nursing teams, is needed to support families considering this future treatment.
Area of Science:
- Pediatric Hematology
- Genetic Medicine
- Patient Advocacy
Background:
- Gene therapy is an emerging treatment for severe childhood diseases.
- Currently, gene therapy is not an approved treatment for hemophilia in children.
- It may offer future therapeutic options, particularly in resource-limited settings.
Purpose of the Study:
- To evaluate parental attitudes towards gene therapy for pediatric hemophilia.
- To assess parental awareness and information needs regarding gene therapy.
- To explore motivations and barriers influencing the consideration of gene therapy.
Main Methods:
- A mixed-methods approach combining online surveys and qualitative focus group interviews.
- Thematic analysis was employed to interpret qualitative data from interviews.
- Survey data provided quantitative insights into parental awareness and interest.
Main Results:
- Most parents surveyed had heard of gene therapy but lacked a deep understanding.
- A significant portion of parents were unaware that gene therapy is not yet available for children with hemophilia.
- Despite awareness gaps, a strong majority of parents expressed willingness to consider gene therapy for their child.
Conclusions:
- A unified communication strategy is essential for disseminating information about gene therapy to hemophilia communities.
- Trusted healthcare providers, especially hemophilia nursing teams, should lead this communication.
- Information must be age-appropriate, factual, and tailored to the needs of children and their families.
Introduction:
Gene therapy is used in life-limiting conditions of childhood. While not a current therapeutic option for children with haemophilia, it may be considered in the future especially for those where access to treatment is limited.
Aim:
To assess the attitudes and opinions of parents of children with haemophilia about gene therapy as a potential future treatment, by understanding their awareness about gene therapy and what they need to know now and in the future; gauging levels of interest in gene therapy for their children; and exploring perceived current motivations and barriers.
Methods:
A mixed methods study with an online questionnaire and in-depth qualitative interviews in focus groups which were analysed using thematic analysis.
Results:
One hundred and fifty-eight participants commenced the online survey; 63 were fully completed (39%). 60 had heard of gene therapy but few (17/60 [28.3%]) felt they had a good understanding. 38/60 (63.3%) respondents did not know that gene therapy is not available for children. However, most held positive views: 53/60 (88.3%) saying they would consider it for their child. In the interviews, participants (N = 10, all mothers) discussed their awareness and understanding of gene therapy and opinions about it for children, including how this should be communicated to the child and parents.
Conclusion:
A coherent, community-wide strategy for communicating information and news about gene therapy should now be provided for children and families living with haemophilia. This should come primarily from trusted haemophilia nursing teams, who can give tailored, age-appropriate, factual advice.
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