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Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease
Colleen A Kelly1,2, Morgan A Paul3, McKenzie Griffin1
1Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.
Insights
Gene therapy for sickle cell disease (SCD) shows promise, but many families face resource barriers. Household material hardship significantly impacts their ability to access potentially curative treatments, highlighting the need for targeted support.
Area of Science:
- Hematology
- Genetics
- Public Health
Background:
- Sickle cell disease (SCD) is a severe inherited blood disorder associated with significant morbidity, reduced quality of life, and premature mortality.
- Gene therapy presents a potential curative approach for SCD, but its complex and resource-intensive nature raises concerns about accessibility.
Purpose of the Study:
- To assess the proportion of families with children diagnosed with SCD who report the capacity to access a hypothetical curative therapy.
- To identify resource-related barriers that impede families' access to advanced SCD treatments.
Main Methods:
- A cross-sectional study was conducted involving 100 families with children aged 2-17 years receiving care for SCD at a quaternary care center.
- A survey collected data on household characteristics, material hardship (food, housing, utilities, transportation insecurity), and perceived ability to access a hypothetical curative treatment.
- Multivariable logistic regression analyzed the association between household material hardship, disease severity, and reported access to curative therapy.
Main Results:
- Among 82 eligible families, 60% reported experiencing household material hardship in the preceding year, despite existing support services.
- Only 40% of parents indicated that their current resources would allow them to access a curative therapy.
- Household material hardship was independently associated with a 95% reduction in the odds of reporting the ability to access curative therapy.
Conclusions:
- Fewer than half of families with children affected by SCD reported the ability to access gene therapy with existing resources.
- Household material hardship affects a significant majority of families and is a key predictor of reduced access to potentially curative SCD treatments.
- As resource-intensive SCD therapies become more available, interventions targeting resource limitations are crucial for ensuring equitable access and treatment receipt.
Background:
Sickle cell disease (SCD) is a highly morbid condition resulting in poor quality of life and early death. Gene therapy offers opportunity for cure, but its resource‑ and time‑intensive nature may limit access.
Procedure:
We conducted a single-center, cross-sectional study to determine the proportion of families with a child with SCD who report the ability to access a hypothetical curative therapy and to identify family-reported resource barriers. A single-timepoint survey was administered to 100 families with children aged 2-17 years receiving sickle cell care at a quaternary care center. The survey queried parent-reported household characteristics and unmet basic needs in food, housing, utilities, or transportation (household material hardship [HMH]) and assessed parent-reported ability to access a hypothetical curative treatment based on a vignette. Multivariable logistic regression examined associations of HMH and disease severity with family-reported access.
Results:
Of the evaluable 82 families, 60% experienced HMH over the prior 12 months despite robust social work involvement (78%) and participation in means-tested benefits (59%). Forty percent of parents reported an ability to obtain curative therapy with current resources. Upon adjustment for key covariates, HMH exposure was independently associated with 95% lower odds of family-reported ability to obtain curative therapy.
Conclusions:
Although gene therapy offers a potential cure for SCD, fewer than half of families reported ability to access it with current supports. HMH exposure affected nearly two-thirds of families and was independently associated with lower odds of access. As highly centralized, resource-intensive treatments expand, resource-targeted interventions are needed to ensure equitable access and receipt.
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