Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
A CRISPR Focus on Attitudes and Beliefs Toward Somatic Genome Editing From Stakeholders Within the Sickle Cell Disease Community
source: Genetics in Medicine
year: 2019
authors: Persaud A, Desine S, Blizinsky K, Bonham VL
summary/abstract:Purpose:
Genome editing holds both tremendous therapeutic promise and significant potential risk. Sickle cell disease (SCD), the most commonly inherited blood disorder, is a frontline candidate for the clinical applications of this tool. However, there is limited knowledge of patient community values and concerns regarding this new technology. This study aims to investigate the perspectives of three key decision-makers (patients, parents, and physicians) toward participation in future CRISPR-mediated somatic genome editing clinical trials.
Methods:
We utilized a mixed-methods approach, involving an educational video tool, two-part survey, and 15 moderated, audio-recorded focus groups, which were conducted in seven U.S. cities.
Results:
Study participants expressed hope that genome editing technology would rechart the course for SCD, but concerns related to involvement burden, uncertainty of clinical outcomes, and equity in access were identified. Major themes emerged from the focus groups: facilitators of, and barriers to, participation in future somatic genome editing clinical trials; information pertinent to the decision-making process; persons from whom participants would seek counsel before making a decision; and recommendations for the research community on meaningful engagement as clinical trials are designed and approved.
Conclusion:
The advent of genome editing has renewed hope for the SCD community, but caution tempers this optimism.
organization: National Institutes of Health, USA; Rush University Medical Center, USADOI: 10.1038/s41436-018-0409-6
read more full text
+myBinderRelated Content
-
Phase 3 study of L-Glutamine in sickle cell disease: Analyses of time to first and second crisis and average cumulat...A Phase 3, double-blind, randomized, pla...
-
Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is SafeAn independent review board has found Gl...
-
Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell DiseaseMemantine, a standard treatment for Alzh...
-
The Clinical Trial Journeyhttps://www.youtube.com/watch?v=iWqQiJeP...
-
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical TrialImara’s investigational oral inhibitor...
-
Gene-Editing Treatment Shows Promise for Sickle Cell DiseaseScientists are seeing promising early re...
-
Researchers raise funds for phase 1 trial to test medical cannabis in sickle cell diseaseThe Sickle Cell Foundation of Georgia ...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.