Perspectives of Sickle Cell Disease Stakeholders on Heritable Genome Editing

Hollister, Brittany M.; Gatter, Mariclare C.; Abdallah, Khadijah E.; Armsby, Alyssa J.; Buscetta, Ashley; Byeon, Yen Ji Julia; Cooper, Kayla; Desine, Stacy; Persaud, Anitra; Ormond, Kelly E.; Bonham, Vence L.

doi:10.1089/crispr.2019.0034

Cited by 13 publications

(13 citation statements)

References 20 publications

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“…The discussion of GT revealed a willingness to participate in an early‐phase clinical trial. Our study confirms observations in the general SCD community for the consideration of GT 30–32 . Historically, African Americans have been reported to have a distrust of the medical community in general, and clinical trials, in particular 33 .…”

Section: Discussionsupporting

confidence: 88%

“…Our study confirms observations in the general SCD community for the consideration of GT. [30][31][32] Historically, African Americans have been reported to have a distrust of the medical community in general, and clinical trials, in particular. 33 Haywood et al 34 report changing attitudes among SCD patients about participation in clinical trials.…”

Section: Discussionmentioning

confidence: 99%

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Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

Sinha

Bakshi

Ross

et al. 2020

Pediatric Blood & Cancer

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Background: Improved outcomes and the availability of clinical trials of hematopoietic cell transplantation (HCT) from alternate donors and genetically modified autologous hematopoietic progenitor cells have expanded the applicability of HCT for sickle cell disease (SCD). To understand the perspective of primary caregivers exploring HCT in the current milieu, we asked the research question "What motivates primary caregivers to decide to consider HCT and to seek, and to attend, an HCT consultation?" Procedures: We conducted qualitative interviews with primary caregivers within one week after a consultation for HCT for SCD. Data were analyzed using open and axial coding stages of grounded theory methodology. Results: We interviewed 29 primary caregivers (26 females, age 29 to 64 [median 42] years). Primary caregivers report of SCD complications in their child included at least one in the last year by 23 (82%), few or none by 8 (28%), and pain on ≥3 days a week by 13 (46%) primary caregivers. Qualitative analysis revealed that primary caregivers, (i) learn about curative options through social networks, social media, and the news media; (ii) seek consultation because of their child's diminished quality of life, recent complications, an imminent major medical decision, or anxiety about future severe complications; and (iii) see gene therapy as a new, less invasive, and more acceptable treatment. Conclusion: Primary caregivers of children with SCD learn about HCT through social networks, social and news media, and explore HCT as a means to prevent SCD complications and help their child live a normal life.

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Section: Discussionsupporting

confidence: 88%

Section: Discussionmentioning

confidence: 99%

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

Sinha

Bakshi

Ross

et al. 2020

Pediatric Blood & Cancer

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“…These advances in genome science and technology have led to potential therapy for sickle cell disease using CRISPR genome editing technology. New genome-based technology for developing therapies presents opportunities for community-based participatory research [ 56 , 57 , 58 ].…”

Section: Resultsmentioning

confidence: 99%

Integrating Datasets on Public Health and Clinical Aspects of Sickle Cell Disease for Effective Community-Based Research and Practice

et al. 2020

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Sickle cell disease (SCD) is a genetic disease that has multiple aspects including public health and clinical aspects. The goals of the research study were to (1) understand the public health aspects of sickle cell disease, and (2) understand the overlap between public health aspects and clinical aspects that can inform research and practice beneficial to stakeholders in sickle cell disease management. The approach involved the construction of datasets from textual data sources produced by experts on sickle cell disease including from landmark publications published in 2020 on sickle cell disease in the United States. The interactive analytics of the integrated datasets that we produced identified that community-based approaches are common to both public health and clinical aspects of sickle cell disease. An interactive visualization that we produced can aid the understanding of the alignment of governmental organizations to recommendations for addressing sickle cell disease in the United States. From a global perspective, the interactive analytics of the integrated datasets can support the knowledge transfer stage of the SICKLE recommendations (Skills transfer, Increasing self-efficacy, Coordination, Knowledge transfer, Linking to adult services, and Evaluating readiness) for effective pediatric to adult transition care for patients with sickle cell disease. Considering the increased digital transformations resulting from the COVID-19 pandemic, the constructed datasets from expert recommendations can be integrated within remote digital platforms that expand access to care for individuals living with sickle cell disease. Finally, the interactive analytics of integrated expert recommendations on sickle cell disease management can support individual and team expertise for effective community-based research and practice.

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“…Recent research indicates that CRISPR has tremendous potential to improve the care of hematologic diseases, such as sickle cell disease and other heritable hemoglobinopathies, as well as autoimmune conditions, such as lupus [35,47,[103][104][105]. But these opportunities must be weighed against potential downstream effects.…”

Section: Expert Opinionmentioning

confidence: 99%

Harnessing the potential of CRISPR-based platforms to advance the field of hospital medicine

McCarthy

2020

Expert Review of Anti-infective Therapy

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Perspectives of Sickle Cell Disease Stakeholders on Heritable Genome Editing

Cited by 13 publications

References 20 publications

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

Integrating Datasets on Public Health and Clinical Aspects of Sickle Cell Disease for Effective Community-Based Research and Practice

Harnessing the potential of CRISPR-based platforms to advance the field of hospital medicine

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