2017
DOI: 10.1007/s12325-017-0587-7
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Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease

Abstract: Introduction: Sickle cell disease (SCD) is a chronic genetic disease with high morbidity and early mortality; affecting nearly 100,000 individuals in the United States. Bone marrow transplantation, the only curative treatment, is available to less than 20% of patients, due to a number of access barriers. Gene transfer therapy (GTT) has shown to be curative in animal models, and is approved for use in humans for early-phase studies at a few centers. GTT would offer a more accessible treatment option available t… Show more

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Cited by 27 publications
(70 citation statements)
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“…Individuals with SCD will now be faced with the decision to participate in a clinical trial that could cure their disease, but also carries uncertain yet potentially significant risks of side effects. To make an informed decision about participation, adults and parents of children living with SCD need access to clear and comprehensible information about the state-of-the-art of genome editing and gene therapies (Strong et al 2017), especially details differentiating general, germline, and somatic therapies for SCD. An ethically designed consent process can enhance a patient's autonomy and invite them to participate in research when it is consistent with their values and personal goals, while also enabling them to advance the frontiers of scientific knowledge.…”
Section: Discussionmentioning
confidence: 99%
“…Individuals with SCD will now be faced with the decision to participate in a clinical trial that could cure their disease, but also carries uncertain yet potentially significant risks of side effects. To make an informed decision about participation, adults and parents of children living with SCD need access to clear and comprehensible information about the state-of-the-art of genome editing and gene therapies (Strong et al 2017), especially details differentiating general, germline, and somatic therapies for SCD. An ethically designed consent process can enhance a patient's autonomy and invite them to participate in research when it is consistent with their values and personal goals, while also enabling them to advance the frontiers of scientific knowledge.…”
Section: Discussionmentioning
confidence: 99%
“…As noted earlier, SCD has a significant impact on the health status of affected individuals (Fernandes, 2017;Ribeil et al, 2017;Strong et al, 2017). Anaemia and pain are the most frequently experienced and most acute health problems associated with the disease.…”
Section: 5mentioning
confidence: 91%
“…It appeared that the construction and reconstruction of an affirmative identity resulted where there was clear congruity between expectations of all in the situation and their experiences. In other words, trust between patients with SCD and healthcare providers appeared enhanced where communication of information was unambiguous (Strong et al, 2017). The illness experience and associated communications were influenced, however, by an uncertainty that was integral to SCD (Brown et al, 2016).…”
Section: Shaping a Reality Around Sickle Cell Disorder 82mentioning
confidence: 99%
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